Tag : Rare Disease

Gastroenterology

Researchers identify genes linked with survival in children with biliary atresia

Newsemia
Scientists at Cincinnati Children’s Hospital Medical Center have identified an expression pattern of 14 genes at the time of diagnosis that predicts two year, transplant-free...
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Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newsemia
James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on...
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A Preclinical Solution to Studying Neurological Diseases

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At SfN 2018, Antti Nurmi explained a case study of how Charles River have used neuroimaging to study Batten’s Disease. Source link...
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Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

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Leadiant Biosciences, Inc. today announced that the Food and Drug Administration has granted approval to Revcovi (elapegademase-lvlr) injection in the U.S. Revcovi is a new...
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Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

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Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today updated positive results from its Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered...
Neurology

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Newsemia
Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase...
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CHOP experts identify new autoimmune disorder along with personalized treatment for young patient

Newsemia
Elijah Patino is a happy, healthy seven-year-old now, but it took a while to get there. For much of his life, he had a mysterious...

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