welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy
FibroGen, Inc., a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD).
“We are pleased to have received Orphan Drug Designation from the FDA for pamrevlumab in the treatment of DMD. There is high unmet medical need for patients suffering from this debilitating disease needing a new treatment option,” said Elias Kouchakji, M.D., Senior Vice President, Clinical Development and Drug Safety. “All 21 non-ambulatory DMD patients in our ongoing phase 2 study with pamrevlumab have completed the first 52 weeks of treatment. We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis. We look forward to the continued development of this investigational therapeutic.”
The Gene-Edited Babies And CRISPR-Cas9 Controversy, ExplainedOutrage continues to grow over the news ...
Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular DystrophyThe aim of this multicentre, prospective...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
Microdystrophin Gene Transfer Study in Adolescents and Children With DMDThis is a randomized, controlled, open-l...
ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne Muscular Dystr...ReveraGen BioPharma, Inc. today announce...
Discovery Could Lead to Improved Therapies for Duchenne Muscular Dystrophyhttps://www.youtube.com/watch?v=TzoqfAak...