Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for ...

Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Learn more about whether Avidity Biosciences, Inc. or TG Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

PTC Therapeutics, Inc. (NASDAQ:PTCT – Get Free Report) has earned a consensus rating of “Hold” from the fifteen research ...

Columnist Shalom Lim is working with Julia Cameron's popular book "The Artist's Way" as a route to develop his creative self, ...

Whether New Hampshire patients nearing death should be able end their own life with the help of a physician was up for debate ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...

Johnny Quintana’s life changed at the age of 19 when he was diagnosed with a rare form of muscular dystrophy called Facioscapulohumeral muscular dystrophy (FSHD).

European regulators are to review Sarepta Therapeutics’ Duchenne muscular dystrophy drug, eteplirsen – a drug that has sparked controversy in the US, where it was approved by the FDA against ...