Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Entrada has secured authorisation from the UK's MHRA to commence ELEVATE-44-201, to evaluate ENTR-601-44, aimed at treating DMD.

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Mammoth Biosciences, Inc., a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced new ...

Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...

Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

RNA interference (RNAi) therapeutics are based on a natural process by which RNA sequences can block the expression of DNA into protein. The RNAi molecule binds to specific messenger ... Golodirsen, ...

And this change is no exception. Comments under a MailOnline article announcing the smart tills claimed it would make shopping a ‘sterile chore’. Others dispaired at the lack of personal ...