California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
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A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...
In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...
Researchers at Washington University have discovered a new genetic disorder affecting protein folding, offering potential new treatment avenues for rare brain conditions. When most people feeling ...
British drugmaker AstraZeneca increased its full-year sales outlook on booming demand for its cancer and rare-disease drugs, and said it plans to invest $3.5 billion in its U.S. business by the end of ...
Caroline Quinn, 25, from Boston, MA, faces unique challenges in love due to a rare immune disease and severe allergies. With ...
Ocaliva received the FDA's accelerated approval in 2016 to treat patients with primary biliary cholangitis, a rare disease ...
The Siegel Rare Neuroimmune Association is a non-profit organization dedicated to the support of people with acute ...
Audrey Tyrrell, from Harwich, Essex, was diagnosed with Usher syndrome, a rare genetic disease that affects both hearing and ...
A young girl diagnosed with a rare genetic condition which means she will lose her eyesight has begun a 'visual wish list' ...
Stand Up To Cancer ® (SU2C) today announced a collaboration with Johnson & Johnson aimed at supporting research testing a combination of two types of targeted therapies, teclistamab and daratumumab, ...
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