Edgewise Therapeutics Inc EWTX released 12-month topline results from the ARCH study, an open-label, single-center study assessing EDG-5506 in adults with Becker Muscular Dystrophy (BMD). EDG-5506 is ...

(RTTNews) - Santhera Pharmaceuticals said that it received positive recommendation from the Scottish Medicines Consortium for use of AGAMREE (Vamorolone) within NHS Scotland for the treatment of ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) is one of the best up and coming stocks to invest in now. On June 26, Edgewise Therapeutics announced positive results from its sevasemten program for Becker ...

Regenxbio Inc (NASDAQ:RGNX) revealed on Wednesday new interim data from two additional patients in the Phase 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy for ...

(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 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Avidity Biosciences Inc. has announced positive data in ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Treatment with EDG-5506 resulted in significant ...

(RTTNews) - Capricor Therapeutics (CAPR), Tuesday announced positive 3-year results from the ongoing HOPE-2 open-label, randomized, double-blind, placebo-controlled extension study with CAP-1002 for ...

The Momentum trial from Sarepta Therapeutics is investigating SRP-5051 (vesleteplirsen) for use among male patients aged 8 to 21 years who have Duchenne muscular dystrophy amenable to exon 51 skipping ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. BridgeBio said Monday that an experimental ...

Liestal, Switzerland, June 22, 2017 - Santhera Pharmaceuticals (SIX: SANN) announces that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive ...