Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
The U.S. FDA announced today its decision to approve AGAMREE (vamorolone), a new steroid for individuals with Duchenne muscular dystrophy 2 years or older. This approval comes largely based on […]
Leading Duchenne Muscular Dystrophy Nonprofit Strengthens Leadership Team and Board Expertise
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for AGAMREE’s approval in Europe.
– GRAND CANYON is the first pivotal study of an investigational therapy for Becker–– CANYON, the initial Phase 2 cohorts, is fully enrolled – BOULDER, Colo.–(BUSINESS WIRE)– Edgewise Therapeutics, Inc. (Nasdaq: […]
See Previous News Release – Initiation of Phase 1 clinical trial marks Entrada’s transition into a clinical company – – Data anticipated in the second half of 2024 – BOSTON, […]
FibroGen announced topline data from their Phase 3 trials of their novel antifibrotic agent, pamrevulmab, a connective tissue growth factor antibody for the treatment for ambulatory individuals with Duchenne. While pamrevlumab was generally safe and well tolerated, the study unfortunately did not meet the primary endpoint. Pamrevlumab also failed to meet its primary endpoint in non-ambulatory individuals earlier this year.
“CureDuchenne is a leader in funding pioneering research aimed at advancing the best possible therapies for DMD patients, and we value their vote of confidence and financial support of our snRNA-mediated exon skipping platform,” said Jim Burns, Ph.D., Locanabio’s chief executive officer.
My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.
