– 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, […]
A Race Against Time: The CureDuchenne Clinic’s Remarkable Efforts Bring Life-Changing Gene Therapy to Young Boy on the Brink of Ineligibility.
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.
In a promising development, the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHNDC) has advanced the nomination of Duchenne muscular dystrophy (DMD) for inclusion in the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth.
– First participant is expected to be dosed in September 2023 with data anticipated in the second half of 2024 – – Cash runway extended through the end of 2025 […]
While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]
CLICK BELOW FOR A FEW WORDS FROM OUR TEAM AND SAREPTA: Sarepta Therapeutics’ gene therapy for Duchenne, SRP-9001, has been granted Accelerated Approval by the FDA for individuals with Duchenne […]
NEW YORK, June 15, 2023 – CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD), today announced a collaborative clinical trial grant to test repurposing of the FDA-approved […]
