JScreen, a national non-profit public health initiative dedicated to preventing genetic diseases and hereditary cancer, educates communities across the United States on the importance of genetic screening for personal and family health. JScreen announced their support for National Cancer Control Month in April.
Biohaven, a clinical-stage biopharmaceutical company that innovates late-stage products for neurological and neuropsychiatric diseases, announced two major growth achievements.
After discussions with the U.S. Food and Drug Administration, Paris-based Ipsen withdrew the company’s New Drug Application (NDA) for palovarotene.
Shares of bluebird bio were down more than 24 percent after the company announced that the U.S. Food and Drug Administration placed a clinical hold on studies of the gene therapy elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).
A study found potential in a novel gene therapy method to help children born with a rare genetic and neurodegenerative disorder, AADC deficiency.
Shares of Vertex Pharmaceuticals fell in trading after the company announced it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study for a rare disease that can lead to complications of the liver and lungs.
In an effort to boost drug discovery for the “toughest drug discovery problems” through the use of machine learning, precision medicines company Relay Therapeutics is acquiring Waltham, Mass.-based ZebiAI with an $85 million upfront payment.
Roche is calling it quits on a late-stage trial of the company’s Huntington’s disease hopeful tominersen, the Swiss drugmaker said on March 22, a blow for sufferers of the rare, debilitating disease with no treatments to stop or reverse it.
Prescription drug watchdog GoodRx compiled a list of the 10 most expensive drugs on the market.
The U.S. Food and Drug Administration approved Sarepta Therapeutics’ Amondys 45 (casimersen) for patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation amenable to exon 45 skipping.