Will Forte, who played MacGruber on Saturday Night Live, and his brother-in-law Doug Modling, who has Huntington's Disease, ...
Australia's Therapeutic Goods Administration (TGA), responsible for approval of the safety, quality, and efficacy of therapeutic goods, has determined that Skyhawk's SKY-0515 orally-administered small ...
Scientific, political pressures colliding at FDA, delaying rare disease treatment for patients who have few or no options, experts said at CNBC Cures Summit.
Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only ...
FDA Commissioner Marty Makary defended the agency’s recent rejections of rare disease drugs and one of his deputies, Vinay ...
Held Type A meeting with FDA to discuss AMT-130 for Huntington’s disease; Company evaluating Phase III development considerations and plans to ...
Data demonstrating superior selectivity, safety and pharmacokinetics of RGT-0474060 were presented at the 21st Annual Huntington’s Disease ...
The biotech company received regulatory determination that its SKY-0515 candidate qualifies for accelerated registration pathway from the Therapeutic Goods Administration.
Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
uniQure held a Type A meeting with the FDA at the end of January and, in a statement released today, said the agency "cannot agree that data from the Phase I/II studies, compared to an external ...
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