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Regenxbio, Duchenne muscular dystrophy and gene therapy
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy for Duchenne muscular dystrophy.
Regenxbio Share Surge 20% on Duchenne Muscular Dystrophy Treatment Trial Data, Expansion
Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on the year.
Regenxbio eyes 2026 filing for Duchenne muscular dystrophy gene therapy
Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy (DMD), following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial.
2h
Parent Project Muscular Dystrophy Culminates 30th Anniversary with Celebratory "Investing in Every Future" Event
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne, commemorated its 30th ...
1h
on MSN
Capricor wins key EU designations for the lead asset
Capricor Therapeutics (NASDAQ:CAPR) announced Wednesday that the European Medicines Agency has issued Orphan Drug and ...
BioSpace
1d
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 ...
3h
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 ...
rheumatologyadvisor
1h
FDA Roundup: Rheumatology Drug Alerts
Throughout 2024, the FDA issued new drug approvals and expanded treatment indications across various rheumatologic diseases.
FierceBiotech
1d
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 ...
Business Insider
2d
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 ...
4d
on MSN
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.
Reuters
1d
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 ...
1d
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 ...
Muscular Dystrophy News
2h
How I experienced inclusion (and didn’t) during my education
Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...
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Related topics
Regenxbio
Food and Drug Administration
RGX-202
Phases of clinical research
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