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Astellas study results are available for ASP0367 (MA-0211) in Pediatric Male patients with Duchenne Muscular Dystrophy (0367-CL-0102)
Read Astellas community letter below:
Solid Biosciences received clearance by the U.S. FDA for an Investigational New Drug (IND) application for SGT-003 for DMD
Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Community Letter: EMBARK Part 1 Topline Results
Sarepta Therapeutics announced topline results from EMBARK
Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for AGAMREE’s approval in Europe.
Edgewise Therapeutics Announces Initiation of GRAND CANYON, a Global Pivotal Study of EDG-5506 in Becker Muscular Dystrophy (Becker)
– 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: […]
Entrada Therapeutics Announces First Participant Dosed in its Phase 1 Clinical Trial of ENTR-601-44
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, […]
A Race Against Time: The CureDuchenne Clinic’s Remarkable Efforts Bring Life-Changing Gene Therapy to Young Boy on the Brink of Ineligibility
A Race Against Time: The CureDuchenne Clinic’s Remarkable Efforts Bring Life-Changing Gene Therapy to Young Boy on the Brink of Ineligibility.
FibroGen Announces Topline Results from LELANTOS-2, a Phase 3 Clinical Study of Pamrevlumab in Ambulatory Duchenne Muscular Dystrophy
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.
DUCHENNE TRAVEL TIPS
DUCHENNE TRAVEL TIPS:
The Golden Rule is Plan Ahead
Contact the airline ahead of time to approve medical or mobility devices.
Request special travel assistance like checking a scooter or power wheelchair.
Coordinate local transportation and other logistics at your destination.
Research ride options and reserve or rent a wheelchair-accessible van in advance.