Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
The American Journal of Managed Care2h
Sarepta Halts Development, Testing of DMD Therapy SRP-5051
Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...
Yahoo Finance7h
REGENXBIO to Host Webcast Discussing Pivotal Program and First Functional Data from the AFFINITY DUCHENNE® Trial of RGX-202
These updates will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy Association 2024 Breakthroughs ... including RGX-202 for the treatment of Duchenne, ...
Solid Biosciences to Present at the Jefferies London Healthcare Conference
CHARLESTOWN, Mass., Nov. 14, 2024 (GLOBE NEWSWIRE) -- (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company ...
Business Insider17h
Optimistic Outlook for Edgewise Therapeutics with Focus on Sevasemten Data Milestones
The company’s focus has shifted toward upcoming data readouts for sevasemten in Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), following positive results for EDG-7500 in ...
Oppenheimer Remains a Buy on Astria Therapeutics (ATXS)
Oppenheimer analyst Jeff Jones maintained a Buy rating on Astria Therapeutics (ATXS – Research Report) today and set a price target of ...
Netflix Is Back In The Documentary Race With A Slew Of Projects, But Competition Is Still Strong
Netflix flexes documentary muscle with eight projects in the running for an Oscar, but National Geo, others have strong ...
FiercePharma3h
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
Le Lézard6h
Satellos Appoints Stephanie Brown to Board of Directors
Satellos Bioscience Inc. ("Satellos" or the "Company"), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, ...
Hosted on MSN4h
RNA Stock Hits Record High on Entering the Cardiac Disease Space
and del-zota for Duchenne muscular dystrophy (DMD). The most advanced candidate in Avidity’s pipeline is del-desiran. Last ...
Le Lézard22h
Quince Therapeutics Provides Business Update and Reports Third Quarter 2024 Financial Results
Examples of forward-looking statements include, among others, statements relating to current and future clinical development of EryDex, including for the potential treatment of Ataxia-Telangiectasia ...