Edgar toddler first ‘bubble boy’ survivor

Edgar toddler first ‘bubble boy’ survivor

State-mandated test at birth identified fatal disease

By Megan Loiselle
Wausau Daily Herald

EDGAR — A 1-year-old Edgar boy has become the first child in the world to be saved from a fatal immunodeficiency disease, just months after Wisconsin became the only state to test for it at birth.

Dawson Bornheimer’s family on Sept. 25 celebrated the first anniversary of a lifesaving bone marrow transplant that treated his Severe Combined Immunodeficiency, or SCID — also known as “bubble boy disease.” The disease is fatal if not treated in the first few months of life.

The state’s pilot program for the screening was funded in part by the Jeffrey Modell Foundation, established by Vicki and Fred Modell in memory of their son who died at age 15. Dawson was honored as the “man of the year” by the foundation at a gala this spring in New York. The screening now is routine for the estimated 70,000 births in the state annually.

“They caught it, and because of that, we get to play with you every day,” his mother, Melissa Bornheimer, said during an interview at her home, as she patted Dawson’s leg.

Twelve days after Dawson was born on June 12, 2008, the Bornheimers received a call saying he had failed the test for SCID — a test they didn’t even know he had been given.

Melissa said at first she thought the test had to be wrong because Dawson seemed fine.

Hours after the call, the Bornheimers took Dawson to the hospital with a fever. He was released with what doctors said was a viral infection. Days later, his belly button became infected so badly that it had to be removed at a hospital in Madison.

In a span of a few weeks in August, Dawson was checked into the Children’s Hospital of Wisconsin in Milwaukee for a blood transfusion as well as surgeries to remove abscesses growing on his neck and to fix a hole in his trachea. Test results showed his bone marrow wasn’t producing white blood cells strong enough to attack infections in his body. The only cure would be a bone marrow transplant.

On Sept. 25, 2008, he received the transplant, with marrow donated from a baby in Germany. Some of Dawson’s vital organs shut down as his body struggled to adjust to the donor’s marrow, but Melissa said his body eventually accepted the bone marrow.

One year after the transplant, Dawson still has 100 percent of his donor’s bone marrow, which means chemotherapy he received last year was successful in eradicating all of his own bone marrow.

Melissa, 35, said the couple had great health insurance through Mike’s work as a material handler at Wausau Supply Co. It allowed the family to focus on Dawson rather than worrying about his $1 million in treatment costs.

Today, Dawson often crawls around the floor and plays at home with his 9-year-old brother, Dylan. Dawson will start walking soon.

His grandmother Ione Bornheimer of Athens said every time she and her husband, Harvey, visit, they see more improvements.

“He’s making big strides,” she said. “It has been very hard on the family but he’s our little miracle baby.”
linkback url: http://www.wausaudailyherald.com/article/20091008/WDH0101/910080625/1981/WDHopinion

‘Olivia’s Fund’ gets barbecue boost

‘Olivia’s Fund’ gets barbecue boost

The Silliker family and supporters are grateful to the community for its support. As a result of that support, Olivia Silliker’s (small photo) future is considerably brighter following a bone marrow transplant courtesy of her four-year-old sister Mackenzie.

Community support

Family and friends of Olivia Silliker are overwhelmed by the community spirit and generosity displayed at a fund-raising barbecue outside Chrissy’s Variety Store.

The recent barbecue raised $1,500, for which the family and supporters wished to express thanks from the bottom of their hearts.

Olivia was born June 9th to parents Mark and Anna and hospitalized August 5th with a diagnosis of ADA SCIDS (Severe Combined Immunodeficiency). In layman’s terms, the rare (one in every two million births) condition means her immune system does not function properly, and a common cold can be deadly

Treatment for ADA SCIDS is a bone marrow transplant.

The immediate family was tested and a perfect match found in Olivia’s four-year-old sister Mackenzie. One week after admission to Sick Kids hospital in Toronto, the transplant took place with the support of a very brave older sister. Mackenzie has since recovered from the procedure and attended her first day of school September 22nd.

Olivia remains in Sick Kids, in an isolation room, which filters air 500 times per hour. To interact with Olivia, her family needs to protect her from germs by using sterile gowns, gloves, hairnet, boots and masks.

It is unknown how long she must be in the hospital, but her family has been told to prepare for a lengthy stay. Olivia’s blood is taken regularly to ensure her white blood cells are increasing and tests are done to determine whether the bone marrow transplant was a success. She will not be released until it is positive that she has developed an immune system and will be able to fight infection on her own.

In the interim, Anna’s desire to remain with her daughter is being challenged by financial realities. Her maternity leave from a subsequently-closed plant (DDM) will come to an end, highlighting the additional financial pressures of living costs and travel expenses exacerbating the family’s natural stress caused by their daughter’s medical challenges.

Fund-raising efforts have been organized, including the barbecue, in order to provide financial assistance for Anna to stay by Olivia’s side through accommodation at Ronald McDonald House in Toronto, as well as with monthly bills and everyday living expenses.

“I would rather sell my house than leave her there all alone,” said Anna.

Additional fundraisers will occur over the next few months, however friends and family are encouraging the public to donate whenever possible to assist the Silliker family.

Donations can be made at St. Mary’s Roman Catholic Church in Tillsonburg. Cheques are to be made payable to St. Mary’s RC Church specifying that the donation is for “Olivia’s Fund” and a tax receipt will be issued.

linkback url: http://www.tillsonburgnews.com/ArticleDisplay.aspx?e=2047035

Scientists Identify Genetic Cause of Previously Undefined Primary Immune Deficiency Disease

Scientists Identify Genetic Cause of Previously Undefined Primary Immune Deficiency Disease

Researchers at the National Institutes of Health have identified a genetic mutation that accounts for a perplexing condition found in people with an inherited immunodeficiency. The disorder, called combined immunodeficiency, is characterized by a constellation of severe health problems, including persistent bacterial and viral skin infections, severe eczema, acute allergies and asthma, and cancer.

The team that made the discovery was led by Helen Su, M.D., Ph.D., at the National Institute of Allergy and Infectious Diseases (NIAID), and included collaborators from NIAID and the National Cancer Institute (NCI). The research is reported in this week’s New England Journal of Medicine.

“NIH clinicians have cared for people with unusual and difficult-to-treat immune disorders for decades,” says NIAID Director Anthony S. Fauci, M.D. “This study exemplifies their commitment to improving the lives of people with these diseases by trying to uncover the causes of these disorders and thereby better understanding how to treat them.”

Combined immunodeficiency is a type of primary immune deficiency disease (PIDD) in which several parts of the immune system are affected. This inherited disorder is characterized by increased susceptibility to bacterial, viral and fungal infections of various organs of the body. In some cases, susceptibility to cancers also may be seen.

There are 150 known PIDDs. Approximately 500,000 people in the United States have been diagnosed with a PIDD, while many more remain undiagnosed.

The NIAID and NCI investigators recognized that certain patients with an undefined form of combined immunodeficiency shared enough clinical features to make it likely that the cause might be a common genetic mutation. Originally, these individuals were thought to have a variant form of hyper-immunoglobulinema E syndrome (HIES), a disorder characterized by increased levels of a class of antibodies known as immunoglobulin E, superficial and systemic bacterial and fungal infections, and atopic dermatitis, also known as eczema.

This newly described group, however, had far more severe eczema than is typical in people with variant HIES.  They also had extensive and difficult-to-manage viral infections of the skin, such as warts, molluscum contagiosum—a type of poxvirus that only infects the skin—and herpes simplex. Some in this group also developed skin cancers, as well as lymphoma of the skin.

“Even though these individuals were diagnosed with a more uncommon form of HIES, they were still considered to have a mystery disease, because they had severe allergies and had developed cancers,” says Dr. Su.

Using a technique called comparative genomic hybridization, a process by which large amounts of DNA is fixed to a computer chip and analyzed for changes in the genes, scientists examined the genes in the tissue samples from five different groups:  the 11 individuals with the unknown immunodeficiencies, people with the variant form of HIES, people with classic HIES, those with other immunological diseases, and healthy individuals.

The researchers discovered that people with the unique form of HIES had mutations in a gene called DOCK8 that led to deletions in parts of the gene. The normal function of DOCK8 is currently unknown.

When compared with healthy individuals, the people with DOCK 8 mutations had fewer CD8 positive T cells, immune cells needed to fight viral infections; fewer antibody-producing B cells; and increased numbers of eosinophils—immune cells associated with allergy.

According to Dr. Su, these findings indicate that DOCK8 is essential for defense against viral infections and for preventing the development of cancer and allergies.

Although further study is required to determine if DOCK8 mutations occur in other people with similar disease symptoms, DOCK8 immunodeficiency syndrome may be a new PIDD. These findings mean that individuals with this rare disease will be able to receive a more accurate diagnosis. Identifying a genetic cause for the disease provided comfort to some of those diagnosed who had battled an unknown immune disease for years, according to Dr. Su.

“The study of inherited disorders and the genetic alterations that are responsible for their complex array of disease symptoms has often resulted in the discovery of causative genes that play a role in cancer initiation,” said NCI Director John E. Niederhuber, M.D. “The disease mutations found in this study in the DOCK8 gene exemplify that kind of important finding.  As with any discovery of a genetic defect, the challenge going forward is to develop a complete knowledge of the cascading pathways of biological function for which DOCK8 is responsible.”

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NIAID conducts and supports research—at NIH, throughout the United States, and worldwide—to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID Web site at http://www.niaid.nih.gov.

NCI leads the National Cancer Program and the NIH effort to dramatically reduce the burden of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI Web site at http://www.cancer.gov or call NCI’s Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).

The National Institutes of Health (NIH)—The Nation’s Medical Research Agency—includes 27 Institutes and Centers and is a component of the U. S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

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Reference:

Zhang et al. DOCK8 mutations underlie a new form of combined immunodeficiency with allergy, hyper-immunoglobulinemia E, lymphocyte dysfunction, viral infections, and cancer. New England Journal of Medicine DOI: 10.1056/NEJMoa0905506 (2009).

linkback url: http://www3.niaid.nih.gov/news/newsreleases/2009/DOCK8.htm

The Second Coming of Gene Therapy

The Second Coming of Gene Therapy
09.02.2009
For years, gene therapy produced tons of hype but no results. Recently, though, new approaches have yielded its first successes: breakthrough treatments for blindness, cancer, and the deadly bubble boy disease.
by Jill Neimark

“For the first two years of her life, my daughter, Katlyn, was knocking on heaven’s door every day,” says Daisy Demerchant, a 26-year-old mom living in Centreville, New Brunswick, just north of Maine. “Two months after she was born she started getting sick, and she never got better.” At six months Katlyn was diagnosed with “bubble boy” disease, formally known as severe combined immunodeficiency (SCID), which robs the immune system of the ability to fight infection. There are many causes of this disorder; in Katlyn’s case it was lack of the enzyme adeno­sine deaminase, or ADA, which rids the body of a natural toxin called deoxyadenosine. When the toxin builds up, it destroys T and B lymphocytes, the body’s infection-fighting immune cells. As a result, Katlyn’s immune cells were dying.

Treatment options ranged from risky to grim. One was a bone marrow transplant, in which imported donor cells could manufacture healthy T cells to fight invading germs. But bone marrow transplants can have lethal complications and often require drugs that further inhibit the patient’s immune system, leaving a window of vulnerability until the transplant kicks in. Another potential treatment involved injections of the ADA enzyme itself. But there was a risk Katlyn would develop antibodies to the drug, rendering it useless. Without any treatment at all, she would simply die.

While weighing their options, doctors put the little girl on protective antimicrobials and sent her to a hospital eight hours from her home. She became another fragile bubble baby sequestered from the world. “My husband quit his job building fire trucks, and we lived with Katlyn in the hospital for 15 months,” Demerchant says. The parents had to wear sterile gowns, booties, masks, and gloves, and the urge to touch their child—let alone hug and kiss her—had to be put on hold.

Just when it seemed as if Katlyn’s life might never improve, science and fate intervened. Her specific condition, called ADA-SCID, had long tantalized researchers seeking to repair genetic defects with a technique called gene therapy. Rare, deadly, and caused by a single gene mutation, it was a perfect proof-of-principle condition for anyone seeking to replace damaged DNA with genes that did the job. With all her troubles, little Katlyn Demerchant had been almost made to order for Fabio Candotti, a senior investigator at the National Human Genome Research Institute at the National Institutes of Health in Bethesda, Maryland.

Before Katlyn showed up at NIH, the doctors there were already well prepared: They had inserted healthy human ADA genes into a modified mouse retrovirus—a type of virus that can enter human cells and transfer new genetic material right into the DNA strands in their nuclei.

Once Katlyn arrived in May 2007, Candotti and his team removed stem cells from her bone marrow and exposed them to the engineered retrovirus, creating a human-virus hybrid. Then they injected the hybrid cells back into Katlyn. Like heat-seeking missiles, the retooled stem cells automatically found their way back home to the marrow. There, they began to specialize, creating all of the secondary or “daughter” cells that such stem cells normally produce—including healthy T cells with functioning ADA genes.

Everybody waited while Katlyn, still stuck inside the bubble, learned to walk on the floor of her sterile isolation room and to play through the protective window with a visiting dog named Toffee. On September 3, blood tests showed Katlyn’s immune system was being populated with robust, functioning T cells. She was so restored, in fact, that her parents were able to take her outside for the first time since she was an infant. “The first day we took her out she was really quiet and a little terrified,” Daisy Demerchant says. “The second time she started running around and asking us a million questions. She’d point to the sun, clouds, leaves, cars, everything imaginable, and ask us what it was. Ever since that day, she has never wanted to stay inside.”

Six months after her gene therapy transplant, Katlyn was so healthy that doctors let her return home to Canada. It can take a year or longer for the immune system to reconstitute itself in full, so Katlyn still takes antimicrobials as a precaution, but today she plays outside, even in the dirt, and is resistant enough to fly on a commercial plane.

The new DNA treatments for Katlyn Demerchant and other bubble babies are nothing short of remarkable, the culmination of a major push to perfect gene therapy for the disease, Candotti says. Across the ocean, in Italy, bubble babies with ADA-SCID are also being cured: A trial led by Alessandro Aiuti, a molecular biologist at San Raffaele Telethon Institute for Gene Therapy in Milan, restored the immune system in eight of ten children, while a ninth had significant improvement.

And bubble babies are far from alone. In Europe and the United States, gene therapists have restored vision in individuals suffering from a rare genetic disorder that inevitably leads to blindness. In Texas, a team has manipulated genes in order to put deadly cancers into complete remission. Building on these successes, gene therapy may soon be used to correct hereditary genetic diseases like cystic fibrosis, hemophilia, and Tay-Sachs and to activate the immune response against a wide variety of infectious diseases and cancers. Gene therapy and its adjuncts may help us trick the body into growing new tissue to rejuvenate arthritic joints, fix injured hearts, and speed the healing of wounds….Continued

link url: http://discovermagazine.com/2009/sep/02-second-coming-of-gene-therapy

Campers aid area toddler

The Jeffery family is giving thanks to their newfound friends, many of whom they’ve never met.

Indeed, most of the Jefferys’ supporters are members of the Bluewater Campers’ Association, which recently presented the St. Joseph’s family with a $7,200 donation, the second of such gifts since the club first heard of the family’s plight three years ago.

“I can’t tell you what this means for our family,” says Dennis Jeffery. “This will help a lot.”

And help is what the Jefferys need as their youngest son, three-year-old André, continues with his valiant battle with Severe Combined Immunodefiency Disease (SCID), an autoimmune disease that leaves him vulnerable to a host of life-threatening illnesses and renders most of his life to the confines of what is commonly known as a plastic bubble.

André was first diagnosed with the disease at the age of four months when the Jefferys brought him to see a Stratford doctor as the baby was covered in a rash that looked like he’d been splashed with scalding water.

André has been in and out of hospitals ever since.

Most recently, the sweet-cheeked young lad with soulful brown eyes, suffered cardiac arrest, his second one due to the havoc the 34 medications medications he takes daily are wreaking on his organs.

André is now at Sick Kids’ Hospital in Toronto where his mom, Sheila, keeps vigil as the rest of the family including seven-year-old sister, Monica, and nine-year-old brother, Donavon, hold down the fort in St. Joseph’s.

Naturally, notes Dennis, the emotional, financial and physical toll of André’s plight is difficult on everyone, especially André’s siblings who must spend a lot of time away from their beloved parents who, for a time, were at André’s bedside 24 hours a day.

“It’s hard to keep everybody together,” notes Dennis. “It’s been a difficult experience. I wouldn’t wish it on my worst enemy.

And when André and his mom are able to return home, the family will once again face the additional expenses of bringing Registered Nurses in to assist with his care. Plus, only 27 of the medications André requires are covered by OHIP.

At a recent meeting at the Bluewater Campers’ meeting room, club members presented the family with the $7,200 donation, as well as $50 gift certificates for Monica and

Donavon and a stuffed animal for André. It was clearly a proud night for members as one organizer put it: “For such a small number of people, this is an amazing amount of money.”

While learning André is currently grappling with kidney problems as he recovers from cardiac arrest, the Bluewater Campers vow to continue to help where they can – even though most of them were meeting Dennis, Monica and Donavon for the first time at the cheque presentation.

“It’s going to help me large,” says Dennis, who notes there are many changes on the horizon for the Jefferys including the possibility that they will have to sell their St. Joseph’s home, a community where both Dennis and Sheila hail from, and move to Toronto to ensure Andre continues to receive the treatment he needs.

Club member Deb Jeffrey, in thanking the campers, note the relatively small park – at 140 trailers – has raised nearly $20,000 in the past three years for the Jefferys and other community-based causes including $3,000 for Habitat for Humanity and $2,500 for the CT Scanner campaign at Alexandra Marine General Hospital in Goderich last year, and about $3,000 for the Jefferys two years ago.

The fundraising events, note organizers, were made possible through a slew of events, including silent auctions and golf tournaments with prizes donated by a vast number of area businesses and community groups. A special cake made each year proves to be a hot ticket item as well as it raised $350 during the first year, $550 the second and $1,400 this year.

linkback url: http://www.clintonnewsrecord.com/ArticleDisplay.aspx?e=1716515

Mother’s tribute to hospital

Mother’s tribute to hospital

(UKPA) – Sep 1, 2009

When it was suggested that her very sick daughter have a new bone marrow transplant procedure at Great Ormond Street Hospital (GOSH), Louise Moseley said she was just relieved that Ffion was not the first.

“I would have been more sceptical if she had been the first but other parents had been brave enough to say yes. She was so poorly we just thought that it had to be done.”

Ffion was just three weeks old when she developed skin problems that were so serious that she was regularly in hospital.

It was initially thought to be dermatitis but when Ffion was transferred to the Singleton Hospital in Swansea it was suggested the condition was likely to be caused by a problem with her immune system.

Ffion was three months old when she was diagnosed with the genetic condition Omenn Syndrome, a severe combined immunodeficiency (Scid), and admitted to GOSH.

“All I heard was ’she needs a bone marrow transplant. It’s a fatal condition’,” said Mrs Moseley, 30, of Pembroke Dock, Dyfed. “It was a complete shock. I’d never heard of it because it’s such a rare condition.”

Too sick to have the chemotherapy needed for a conventional transplant, Ffion was given the new procedure using antibodies to prepare her for the transplant which she had aged five months.

Although she suffered complications afterwards, including Graft Versus Host Disease (GVHD) where the body attacks the transplanted tissue, Ffion was well enough to go home three months later and now only needs an annual check up.

“Not only did the BMT save Ffion, but her skin is now beautiful. You’d never know she is the same child that was so desperately ill in the past,” Mrs Moseley said.

linkback url: http://www.google.com/hostednews/ukpress/article/ALeqM5im-K_CuOAjzgPmt75ziH1skVSAVA

‘She’s our little miracle’

‘She’s our little miracle’

Published Date: 17 July 2009
Dressed in pink from head to toe, two year-old Eva McLaughlin skips across the kitchen floor pushing a yellow toy shopping trolley.
“I’m going to the Spar,” chirps the blonde-haired Magilligan girl.
Seconds later, she sits by her pink toy piano and tells mum Cathy: “I’m going to play a song.”

Moments later Eva is off again, this time down the hall chatting.
Anyone meeting Eva for the first time would think she is just an ordinary toddler, full of energy and into everything.
But Eva is anything but ordinary.

“She’s a miracle, she really is,” says Cathy.

Eva was born with severe combined immune deficiency syndrome (SCID), a condition which means she had no white T blood cells which provide natural resistance against infection. Without these, infections spread and can be fatal.

At first, mum Cathy and dad Ryan had no reason to suspect anything was wrong with Eva but when their baby girl couldn’t shake coughs and infections, they knew something was wrong.

“It just wasn’t shifting,” recalls Cathy, explaining the weekly trips to the doctor.

On December 5, 2007, Eva became very ill, struggling for breath. The tot had severe pneumonia.

She was taken to the Royal Victoria Hospital in Belfast for a week where she was kept alive by a ventilator supplying her with life-saving oxygen.

“It was horrific, just complete panic,” says Cathy. “It was so hard to see and look at her in intensive care. She was just lying there.”
Miraculously, Eva was able to come off the ventilator within days. She was taken to Newcastle General Hospital for a bone marrow transplant after a match for her was found by the Anthony Nolan Trust in the USA.
“It was the only chance she had. The bone marrow transplant was the only thing she had to keep her alive,” says Cathy.

Prior to the transplant, Eva underwent eight days of gruelling chemotherapy.

“It was when her hair fell out and I picked it up from the pillow; that was just horrific,” says Cathy.

Just one day after her first birthday, Eva had a rest day. The next day she had the transplant and Cathy and Ryan were told they could not touch their child.

“They said if you kiss that child it could kill her,” recalls Cathy. “It was awful but it needed to be done but knowing I couldn’t even kiss my own daughter was hard.”

When Eva was in hospital, she lived in a tightly-controlled, 8ft x 8ft “bubble” to avoid threats to her non-existent immune system.
Almost 10 stressful months later, Eva and her parents were finally ready to go home.

“Eva was so near death and I wouldn’t wish it on anyone,” says Cathy, reflecting on the ordeal. “The doctors here and in Newcastle are miracle workers. What they did for Eva was unbelievable. It’s just unreal, just the most unbelievable experience. We handed our daughter over to the doctors and said: ‘Please cure her’.”

The trip home was an emotional one for Cathy and Ryan but the close-knit community lined the country road where they lived, wishing them well.

“Everyone was cheering and waving,” says Cathy. “Magilligan is a brilliant community. We had a brilliant reception.”

‘Lucky’

Cathy and Ryan travel to Newcastle on Monday for a check-up on Eva’s progress. They are hoping for an easing of restrictions on things she hasn’t been able to do before, like go to the beach or the zoo.
“The way Eva looks now compared to before is unreal,” says Ryan. “People can’t believe how she’s come on, running about like any other wee child. Looking back on it now with some perspective, you think, ‘We were lucky’.”

Cathy adds: “She’s a miracle, she really is.”

The family want to raise as much awareness as possible for the Anthony Nolan Trust and the Bubble Foundation and with the help of their close knit community have already raised almost £6,000 for research.
“Thank God Eva is here, and every day she’s going from strength to strength. It is a miracle,” adds Cathy.

For more information or to make a donation, go to www.anthonynolan.org.uk and www.bubblefoundation.org.uk

linkback url: http://www.derryjournal.com/journal/Shes-our-little-miracle-.5469463.jp

Baxter and Halozyme Announce Completion of Patient Enrollment in Phase III Pivotal Trial of GAMMAGARD LIQUID™ with rHuPH20 Enzyme

Baxter and Halozyme Announce Completion of Patient Enrollment in Phase III Pivotal Trial of GAMMAGARD LIQUID™ with rHuPH20 Enzyme

(Nasdaq:HALO) today announced completion of patient enrollment in the Phase III pivotal study of GAMMAGARD LIQUID [Immune Globulin Intravenous (Human) 10%] (also known as KIOVIG outside of the United States) with rHuPH20 (recombinant human hyaluronidase enzyme) for the treatment of primary immunodeficiency disorder : (PID). Patients will receive monthly
subcutaneous (SC) injections of Halozyme’s rHuPH20 with Baxter’s GAMMAGARD LIQUID : .

“The achievement of complete Phase III enrollment is an important milestone of Baxter’s work with Halozyme to offer patients more advanced treatment options,” said Hartmut J. Ehrlich, M.D., vice president of Global Research and Development for Baxter’s BioScience business.

“I am pleased with the efficiency and dedication Baxter has demonstrated toward our collaboration and their exemplary ability to manage and carry out the clinical objectives,” stated Jonathan Lim, M.D., Halozyme’s president and CEO. “Completion of patient enrollment in this Phase III registration study marks a significant and timely accomplishment for the GAMMAGARD LIQUID-rHuPH20 development program.”

This Phase III clinical trial is a prospective, open-label, non-controlled design underway in 15 centers in the U.S. and Canada. The study will evaluate the safety and efficacy of GAMMAGARD LIQUID administered SC with rHuPH20 in the prevention of acute serious bacterial infections over 12 months and will also assess pharmacokinetic parameters of SC GAMMAGARD LIQUID with rHuPH20 compared to intravenous (IV) administration of GAMMAGARD LIQUID alone. Patient Quality of Life (QOL) parameters will also be measured. Subcutaneous administration of GAMMAGARD LIQUID with rHuPH20 in this investigational study will determine if it will allow PID patients to receive a full monthly dose in a single injection site in the home setting. GAMMAGARD LIQUID is currently only approved for IV administration. Additional information about this Phase III study can be found at clinicaltrials.gov : .

About GAMMAGARD LIQUID

GAMMAGARD LIQUID is indicated for the treatment of primary immunodeficiency disorders associated with defects in humoral immunity.

These include but are not limited to congenital X-linked agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

Important Safety Information

GAMMAGARD LIQUID is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human).

Patients with severe selective IgA deficiency (IgA < 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction.

Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. IGIV products should be administered at the minimum concentration available and the minimum rate of infusion practicable in at risk patients.

GAMMAGARD LIQUID is made from human plasma. It may carry a risk of transmitting infectious agents, e.g. viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent. Some viruses, such as B19V or hepatitis A, are particularly difficult to remove or inactivate.

GAMMAGARD LIQUID should only be administered intravenously.

Immediate anaphylactic and hypersensitivity reactions are a remote possibility.

IGIV products can contain blood group antibodies which may act as hemolysins and induce in vivo coating of red blood cells with immunoglobulin causing a positive direct antiglobulin reaction and, rarely, hemolysis. Hemolytic anemia can develop subsequent to IGIV therapy due to enhanced red blood cell sequestration.

There have been reports of noncardiogenic pulmonary edema (Transfusion Related Acute Lung Injury [TRALI]) in patients administered IGIV.

Thrombotic events have been reported in association with IGIV. The potential risks and benefits of IGIV should be weighed against those of alternative therapies.

Aseptic meningitis syndrome (AMS) has been reported to occur infrequently in association with GAMMAGARD LIQUID treatment.

Discontinuation of IGIV treatment has resulted in remission of AMS within several days without sequelae.

For full prescribing information, please visit: www.baxter.com/products/biopharmaceuticals/downloads/gamliquid_P .. :

About Baxter

Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, infectious diseases, kidney disease, trauma, and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.

About Halozyme Therapeutics, Inc.

Halozyme is a biopharmaceutical company developing and commercializing products targeting the extracellular matrix for the endocrinology, oncology, dermatology and drug delivery markets. The company’s portfolio of products and product candidates is based on intellectual property covering the family of human enzymes known as hyaluronidases and additional enzymes that target the extracellular matrix. Halozyme’s Enhanze ™ Technology is a novel drug delivery platform designed to increase the absorption and dispersion of biologics. The company has key partnerships with Roche to apply Enhanze Technology to Roche’s biological therapeutics for up to 13 targets and with Baxter BioScience to apply Enhanze Technology to Baxter’s biological therapeutic compound, GAMMAGARD LIQUID ™ . The product candidates in Halozyme’s research pipeline target multiple areas of significant unmet medical need. For more information visit www.halozyme.com : .

Safe Harbor Statement

In addition to historical information, the statements set forth above include forward-looking statements (including, without limitation, statements concerning the GAMMAGARD-rHuPH20 clinical development program) that involve risk and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. The forward-looking statements are also identified through use of the words “believe,” “enable,” “may,” “will,” “could,” “intends,” “estimate,” “anticipate,” “plan,” “predict,” “probable,” “potential,” “possible,” “should,” “continue,” and other words of similar meaning. Actual results could differ materially from the expectations contained in forward-looking statements as a result of several factors, including regulatory approval requirements and competitive conditions. These and other factors that may result in differences are discussed in greater detail in each company’s reports on Forms 10-K, 10-Q, and other filings with the Securities and Exchange Commission.

Baxter Media ContactsChris Bona, 847-948-2815Laura
Grossmann, 847-948-3026orBaxter Investor ContactsMary
Kay Ladone, 847-948-3371Clare Trachtman, 847-948-3085orHalozyme
ContactRobert H. Uhl, Senior Director, Investor Relations,

858-704-8264 ruhl@halozyme.com : mailto:ruhl@halozyme.com

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