Council leader backs campaign to help sick baby

August 10, 2010

Council leader backs campaign to help sick baby

By RACHEL HAWTHORN
THE leader of Portsmouth City Council today called on city residents to join the bone marrow donor register.
Councillor Gerald Vernon- Jackson is the latest person to pledge his support for our campaign to find baby Jasmine Andrew a bone marrow match.

As soon as he heard about the three-month-old’s plight he said he would join the register himself and actively try to recruit people as well.

Cllr Vernon-Jackson said: ‘It seems the least I can do.

‘I’ve carried an organ donor card round for years. What I don’t need, they can have. This is the same. ‘ He added: ‘I will be encouraging everyone I talk to join the register now.

‘The more people we can get to sign up to donate, the better.

‘This is something I really hope lots of people will do.’

The News launched a campaign last week to help tiny tot Jasmine, who has a rare and potentially life-threatening immune system deficiency called Severe Combined Immunodeficiency (SCID). She is constantly vulnerable to infections and viruses and the only cure for her condition is a bone marrow transplant.

Her family made a desperate plea for readers of the News to join the register and see if they are a potential match.

Cllr Vernon-Jackson is now joining the likes of Linvoy Primus and pop duo Same Difference, in pledging his support. He is also helping us to find a venue for a special session in Portsmouth for readers to attend and become donors.

‘I will do whatever I can to help,’ said Cllr Vernon-Jackson.

Jasmine is still at Great Ormond Street Hospital in London being treated by specialists.

She is still trying to recover from a bout of gastroenteritis.

Meanwhile, The News is working with Anthony Nolan – a charity which specialises in finding bone marrow donor matches, to organise sessions across the area.

During the sessions people will be asked to give saliva samples. Dates and venues of sessions will be announced soon.

linkback url: http://www.portsmouth.co.uk/newshome/Council-leader-backs-campaign-to.6464949.jp

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Linvoy steps in to help in Jasmine’s bone marrow plea

August 6, 2010

Linvoy steps in to help in Jasmine’s bone marrow plea

By Unknown
FOOTBALL legend Linvoy Primus is proving he’s a hero off the pitch as well as on it.
The former Pompey favourite is set to join the bone marrow donor register in a bid to help baby Jasmine Andrew.

The three-month-old has a potentially life-threatening immune system deficiency and the only cure is a bone marrow transplant.

As soon as Linvoy heard about the tot’s plight he jumped at the chance to help her and immediately pledged his support for our campaign to find her a bone marrow match.

And now the football star is urging readers of The News to do the same.

He said: ‘I’m more than happy to join the register.

‘If you’ve got an opportunity to help someone, you’ve got to take that. You could literally be a life saver here.

‘I’d definitely encourage News readers to join the register too.’

The News launched a campaign earlier this week to help find Jasmine a bone marrow match.

The tot was born with Severe Combined Immunodeficiency (SCID) and is constantly at risk of infections.

Anthony Nolan – a charity which specialises in finding matches – is backing our campaign to help her.

Together we are set to organise sessions in the area for people to attend and join the register. Linvoy will be joining us at one.

During the events, Anthony Nolan representatives will be on hand to take saliva samples from those willing to join the register.

That sample will then be sent to a laboratory in London where the tissue type will be noted and the person added to the donor register.

If you are found to be a match for Jasmine, or for any other patient in need of a transplant, Anthony Nolan will then contact you.

Linvoy said: ‘I think when you realise how easy and simple it is to do and how effective and life changing it can be, you just can’t say no.’

Details of the bone marrow donor sessions we are organising will be announced soon.

In the meantime, Anthony Nolan are inviting people to sign up online or by calling the charity.

linkback url: http://www.portsmouth.co.uk/newshome/Linvoy-steps-in-to-help.6462053.jp


Secretary of Health and Human Services Announces Addition of SCID to National Newborn Screening Standards

May 25, 2010

Secretary of Health and Human Services Announces Addition of SCID to National Newborn Screening Standards

TOWSON, Md., May 25 /PRNewswire/ — On May 21, 2010, Kathleen Sebelius, Secretary of Health and Human Services (HHS) announced the addition of Severe Combined Immunodeficiency (SCID) — commonly known as bubble boy disease — to the core panel of 29 genetic disorders — as part of her recommendation to adopt the national Recommended Uniform Screening Panel.  The Secretary made her announcement in a letter to Dr. Rodney Howell, Chair of the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC). SCID is the first nominated condition to be added to the core panel of disorders.

SCID is a primary immunodeficiency disease.  Affected infants lack T lymphocytes, the white blood cells that help resist infections due to a wide array of viruses, bacteria and fungi.  Babies with SCID appear healthy at birth, but without early treatment, most often by bone marrow transplant from a healthy donor, these infants cannot survive.

The Immune Deficiency Foundation (IDF), the national patient organization for persons with primary immunodeficiency diseases, applauds the action by Secretary Sebelius for including SCID in the new national standards.  “The addition of SCID to the national newborn screening standards is a momentous step forward for the primary immunodeficiency community,” said Marcia Boyle, President & Founder of IDF.  “The IDF has strongly supported and worked tirelessly toward this goal for years.  It is imperative that we sustain this momentum by establishing newborn screening programs in all 50 states.”

“Although this recommendation has been in development for two years,” said Dr. Amy Brower, parent, researcher and former SACHDNC committee member, “it may take several more years to implement screening in all 50 states and US territories.  We must work to quickly implement the widespread adoption of testing and treatment in all of the states.”

“As the parent of a child who was diagnosed with SCID only after he became critically ill,” said Barb Ballard, a member of the IDF Board of Trustees, “I am immensely pleased with the action taken by Secretary Sebelius.”

“Our goal is to have Newborn Screening for SCID passed in all 50 states,” said Heather Smith, co-founder of SCID Angels for Life, who lost her six-month-old son, Brandon, to this devastating disease.

For more information, please contact IDF at 800-296-4433, or idf@primaryimmune.org.  Or visit the IDF website at www.primaryimmune.org.

SOURCE Immune Deficiency Foundation

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RELATED LINKS
http://www.primaryimmune.org

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Bubble baby in charity funds role

March 2, 2010

Bubble baby in charity funds role

02 March 2010 By David Coates

A little girl who lived in isolation for the first 13 months of her life after being born without an immune system, is now the face of the charity which gave her a second chance. To see Elle Heim-Sarac bouncing around her grandmother’s living room, it is difficult to believe she is anything other than a normal five-year-old girl.

But just over four years ago she was close to death and some of the country’s leading children’s doctors were baffled by the illness which was slowly destroying her.

One occasion even saw doctors advise her mother to get the tot baptised, so convinced were they that she would not live more than a few days.

“It was just every mother’s worst nightmare,” says her mum Cyndi, 22. “I struggle to remember one day from the next. It was just a constant battle to keep her alive.

“All we knew was that she was dying, all her hair was falling out, her skin was shrivelling up, she looked like a Third World child.”

But at three months old she was seen by a visiting paediatrician at Manchester’s Pendlebury Children’s Hospital, who diagnosed Severe Combined Immunodeficiency (SCID), a very rare condition resulting from a gene defect which had left Elle with no immune system.

She and Cyndi left their home in Warton, near Preston, to go to the Children’s Bone Marrow Transplant Unit in Newcastle, one of only two centres in Britain with a hope of saving her.

Her grandmother Sherry, 50, who still lives at the family home, remembers: “Effectively, she had so few cells that those she had to fight infection had recognised her body as the infection and were attacking that.

“They said they could not do a bone marrow transplant on her because she was too poorly, so a stem cell transplant was her only hope. We waited and waited for a donor and one never came.

“Then there was one from Milan, Italy, which only had seven of the 12 matches they needed. It was a real mismatch but the doctors decided they could not wait any longer.”

What followed was weeks of aggressive chemotherapy on the tiny six-month-old to kill off her old cells.

Then there was a major operation to give her new cells which would develop into a new immune system.

But it was a waiting game with Elle living in a sterile “bubble” to protect her from any kind of germs.

Measuring eight feet square, the bubble became Elle’s “home” for the duration of her treatment as even the common cold passed on by her mother’s kiss could have been fatal.

It was during this time that the family got to know the Bubble Foundation, a charity set up to help keep these £2,000-a-day facilities running and provide halfway houses for mothers and babies, plus social care and educational support.

The survival rate of the unit has increased from 50 per cent to 80 per cent in the past five years.

Cyndi says: “I just don’t know what I would have done without them, I would not have been able to do it without them.”

Now five-and-a-half, Elle will join her mum and grandmother at their first fund-raising event for the charity, a family fun day at the Catherine Beckett Community Centre in Deepdale Road, Preston, on Sunday.

Stretched across the outside of that building will be huge poster of Elle as a baby with the words, When A Kiss Can Kill.

As the ever-smiling youngster insists on being asked a question too, after seeing her mum and grandmother take centre stage in the interview, I ask what she would like to say to the charity which helped her get better. She flashes a toothy grin and says: “Thank you.”

The fun day, including a bouncy castle, climbing wall and face painting, runs from 11am until 3pm. For further details or to donate to the Bubble Foundation e-mail: ssarac@uclan.ac.uk

linkback url http://www.lep.co.uk/news/Bubble-baby-in-charity-funds.6113569.jp 


Immune Deficiency Foundation Praises ACHDNC Recommendation for SCID Screening in Newborns

January 27, 2010

Immune Deficiency Foundation Praises ACHDNC Recommendation for SCID Screening in Newborns

WASHINGTON, Jan. 25 /PRNewswire/ — On January 21, 2010, the Advisory Committee on Heritable Disorders in Newborns and Children voted unanimously to add screening for Severe Combined Immune Deficiency or SCID — commonly known as bubble boy disease — to the core panel for universal screening of all newborns in the United States.

SCID is a primary immunodeficiency; affected infants lack T lymphocytes, the white blood cells that help resist infections due to a wide array of viruses, bacteria and fungi. Babies with SCID appear healthy at birth, but without early treatment, most often by bone marrow transplant from a healthy donor, these infants cannot survive.

The Advisory Committee’s policy recommendation will now be presented to Kathleen Sebelius, Secretary of Health and Human Services. Ms. Sebelius has 180 days to consider and respond to the committee’s proposal.

Jennifer Puck, Professor of Pediatrics at the University of California, San Francisco and an expert in the field, nominated SCID for consideration by the Committee after pioneering development of a test that can detect SCID in the dried blood spot filter cards that are currently collected from all babies to screen for a variety of inborn disorders.

“The Advisory Committee has taken a momentous step forward for the primary immunodeficiency community,” said Marcia Boyle, President & Founder of the Immune Deficiency Foundation (IDF), the national patient organization for persons with primary immunodeficiency diseases. “The IDF has strongly supported and worked tirelessly toward this goal for many years. But it is imperative that we sustain this momentum by working to establish newborn screening programs in all 50 states.”

The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) was chartered in February 2003 to perform evidence-based reviews and advise the Secretary regarding application of new screening tests, technologies, policies, guidelines and standards for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders. SCID is the first new disease to be recommended for addition to the federal uniform core-screening panel by the evidence-based Committee review process.

“Although this recommendation has been in development for two years,” said Dr. Amy Brower, parent, researcher and former ACHDNC committee member, “and it may take several more years to implement screening in all 50 states and US territories, we must work to reduce the time from this recommendation to the widespread adoption of testing and treatment.”

In 2004, Duke University Professor of Pediatrics and Immunology, Dr. Rebecca H. Buckley — currently the chair of the Immune Deficiency Foundation’s Medical Advisory Committee and an internationally known expert on SCID —  testified before the ACHDNC (to which she was subsequently appointed in 2007).

Dr. Buckley informed the panel that SCID is a pediatric emergency. If a baby with SCID receives a bone marrow transplant in the first 3.5 months of life, the survival rate can be as high as 94 percent. However, the survival rate drops to less than 70 percent for infants who are transplanted after that age. The main causes for the drop in survival rate are serious infections babies with SCID develop prior to transplantation.

“As the parent of a child who was diagnosed with SCID only after he became critically ill,” said Barb Ballard, a member of the IDF Board of Trustees, “I have to say I am immensely pleased with the recommendation of the committee.”

In addition to the IDF, the Jeffrey Modell Foundation and members of the public whose children were affected with SCID argued in favor of SCID screening. One child born in Wisconsin, where pilot SCID screening is ongoing, was diagnosed at 9 days of age and successfully cured by timely bone marrow transplantation, while another child born in Oregon, where screening does not take place, developed failure to thrive and multiple infections and did not survive.

“Our goal is to have Newborn Screening for SCID passed in all 50 states,” said Heather Smith, co-founder of SCID Angels for Life, who lost her six-month-old son, Brandon, to this devastating disease.

“We have already lost way too many children to this disease,” Ms. Smith concluded, “and newborn screening is the only way we can put an end to the pain and suffering these children are forced to endure.”

The Immune Deficiency Foundation is the national patient organization dedicated to improving the diagnosis, treatment and quality of life of persons with primary immunodeficiency diseases through advocacy, education and research. For information on this media release, please contact Marcia Boyle, president, at 410-321-6647 or idf@primaryimmune.org. For further information please visit the IDF website at www.primaryimmune.org, or call IDF at 800-296-4433.

SOURCE Immune Deficiency Foundation

RELATED LINKS
http://www.primaryimmune.org

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DellaCamera Claims Enzon CEO Can (Almost) Never Be Fired

June 10, 2009

DellaCamera Claims Enzon CEO Can (Almost) Never Be Fired

DellaCamera Capital gathered what it claimed was more than 50 percent of Enzon’s shareholders into a hotel conference room in Midtown New York today to make the case for staging a coup against CEO Jeff Buchalter.

The presenter, DellaCamera research chief Richard Mansouri, dropped some interesting new nuggets into the debate over whether Buchalter should be removed by his shareholders (the stock is down by more than 50 percent over the last five years). Among them:

* Buchalter’s contract requires an unusual 12 months’ notice if the company wants to fire him — and as he’s also contractually required to be Enzon’s board chairman, it is thus virtually impossible for the board to remove him. “We’ve never seen anything like this,” Mansouri said. While dual CEO/chairman roles often create unaccountable “imperial” bosses with built-in conflicts of interest, “The fact that it is contractually mandated is even worse,” he said. Buchalter gets about $5 million a year in compensation even though his company only makes $197 million in revenues.
* Buchalter announced he was exploring the sale of his specialty pharma division in August 2008, and then concluded that effort with no sale in November. No explanation or detail has been offered as to exactly why that effort failed, Mansouri said. “We are not at all satisfied with this lack of information.”
* Enzon has gone through $55 million in “other” R&D costs on “discontinued” products since 2005, and that “other” expenses are the company’s largest R&D cost.

“Something is wrong. Something is very wrong here,” Mansouri concluded.

The audience was subjected to this PowerPoint slideshow, much of which rehashed the case for change that Ralph DellaCamera’s (pictured above) group previously made in a June 1 SEC filing. Get BNET’s take on the back story here.

The chief weakness in the DellaCamera case is that the group won’t say who it thinks should replace Buchalter (pictured below) and nor will it reveal what it believes the company’s strategy should be. “We don’t have any other agenda,” Mansouri said.

This hole will become more important as it gathers shareholder support, because despite its flaws Enzon isn’t a complete disaster: It made $6 million in profit on $48 million in revenue in Q1, both small increases from the year before. Shareholders faced with a stay-the-course plan from Buchalter and no plan at all from DellaCamera might be forgiven for sticking with the devil they know.

In the wings is Carl Icahn, who owns 7.8 percent of Enzon. Icahn just succeeded in putting two supporters on Biogen’s board and two on Amylin’s board. He already has one guy on Enzon’s board.

The event set the stage for an exciting battle, but don’t get too excited: When pressed by BNET, Mansouri all but admitted that the historical success rate of shareholder votes to remove CEOs is zero.

linkback url: http://industry.bnet.com/pharma/10002625/dellacamera-claims-enzon-ceos-contract-says-he-can-almost-never-be-fired/


David’s Dream Run Moves to Bubble Boy’s Hometown

April 17, 2009

David’s Dream Run Moves to Bubble Boy’s Hometown

SHENANDOAH, Texas — David Vetter was twelve years old when he died in 1983 of severe combined immune deficiency (SCID). This young boy from Shenandoah, Texas was known to the world as “the Bubble Boy” and lived his entire life inside a totally germ-free environment. In honor of his memory, the annual David’s Dream Run and Walk was established to help raise money for the David Center at Texas Children’s Hospital’s Allergy and Immunology Clinic, which provides diagnosis and treatment to children with allergy, asthma and primary and secondary immune dysfunction, including HIV/AIDS. The event will be held on Saturday, May 9, 2009.

This year for the first time, the event will be held in David’s hometown. It will start in the parking lot of the new Woodforest Bank Stadium and adjacent natatorium located in Shenandoah behind the Portofino Shopping Center and proceed along David Memorial Drive before returning to the finish line at the stadium. The facilities were opened last year as part of the Conroe Independent School District athletic facilities.

David’s sister, Katherine Vetter Canion expressed the town’s appreciation when she learned the event specifically designed for children and families would be held in Shenandoah. “It’s a home coming we’re so very proud of,” she stated. “The people who knew my brother best and who for years protected his privacy and last name can now be a part of his incredible legacy. It’s very humbling for my mother, father and me and our families, and we are so grateful for the work of all the folks at Texas Children’s Hospital. They will always be very special to us,” she said.

Since 1994, David’s Dream Run has raised over $260,000 for Texas Children’s Hospital. Last year, more than 700 participants and dozens of volunteers took part in the David Dream Run which will consist of both a one mile walk and a 5K run. It is specifically designed for the participation of families, as well as seasoned runners.

This year’s organizing committee consists of Linda Woods, Director of Shenandoah Convention & Visitors Bureau; Lee Allen, Principal of the David Elementary School in The Woodlands; Michelle Barrell and Melissa Wolsky, race volunteers for many years; Brian Carlisle of Shenandoah Police Department; and Katherine Vetter Canion, David’s sister.

All contributions are tax deductible and 100 percent of the funds raised will be donated to the David Center. For more information on how to participate in the David Dream Run and Walk, telephone 281 381-7229 or visit the Web site at the link below.
Related Links:
David’s Dream Run Web site


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