The latest health news
On Friday, the Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee will meet to discuss the Biologics License Application for Sarepta Therapeutics’ gene therapy delandistrogene moxeparvovec for …
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LONDON — Using genome sequencing greatly expanded the number of diagnoses researchers could provide for children with developmental disorders from thousands of families across the United Kingdom and Ireland, researchers …
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My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease …
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Major advances in the study and treatment of rare diseases occurred in 2022, from a better understanding of just how many of these diseases exist to the start of new …
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This month marks the 40th anniversary of the Orphan Drug Act. Since it was signed into law in 1983, the FDA has approved more than 1,100 treatments for rare diseases. …
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At a far distant point in Earth’s ancient past, two separate, single-celled life forms — an archaeon and a bacteria — became one in an act either of symbiosis or …
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Over the past few years, the biopharmaceutical industry has revved up efforts to diversify clinical trials. But clinical trials for rare diseases are still too often homogeneous. Rare disease experts …
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In the spring of 1981, a geneticist from Indianapolis and a neurologist from Chicago got in a car and for three days they crisscrossed the yellowing cornfields of Iowa. Every …
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Rep. Robin Kelly admits that the word “diversity” has scared some of her Republican colleagues away from her effort to improve representation in clinical trials. “It’s been a little tough …
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