Capricor Therapeutics Granted Orphan Drug and ATMP Status for Deramiocel by European Medicines Agency
Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular ...
BioSpace1d
Regenxbio Gears Up to Challenge Sarepta in DMD as Gene Therapy Advances to Pivotal Studies
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...
FierceBiotech1d
After seeing improved muscle function in phase 1/2 trial, Regenxbio launches pivotal DMD study
Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...
REGENXBIO INITIATES PIVOTAL PHASE OF AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY AND REPORTS POSITIVE FUNCTIONAL DATA
REGENXBIO Inc. (Nasdaq: RGNX) today announced that AFFINITY DUCHENNE®, the multi-center, open-label trial of RGX-202, a ...
BioSpace1h
RFK Jr.’s HHS Nomination, Biogen’s Lupus Win and Leqembi Struggles, Neurogene’s Rough Month, More
Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.
Business Insider2d
Regenxbio initiates pivotal phase of Affinity Duchenne trial of RGX-202
a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety ...
Reuters1d
Health Rounds: Vaccine shows early promise against aggressive breast cancer
Elevidys, a gene therapy from Sarepta Therapeutics (SRPT.O), opens new tab approved in the United States last year for ...
I Just Watched The Remarkable Life of Ibelin, And I Was So Moved By How Role-Playing Games Can Empower People with Disabilities
It’s easy for those who play or watch the best movies based on video games to want to be immersed in that fictional world.
Promising Initial Data and Favorable Regulatory Pathway Support Buy Rating for RegenXBio’s RGX-202 in DMD Treatment
Analyst Gena Wang from Barclays maintained a Buy rating on RegenXBio (RGNX – Research Report) and keeping the price target at $50.00.Don't Miss ...
Benefit held for boy with aggressive form of Muscular Dystrophy
DICKSON CITY, Pa. — The Eagle Hose Company in Dickson City on Saturday, November 16 hosted a benefit for the Hetzel family as ...