Sarepta Therapeutics (NASDAQ:SRPT) presented preliminary clinical data from ongoing phase I/II studies of two investigational ...

Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Armed with $68 million in Series B funding, Epicrispr Biotechnologies (Epic) is initiating human trials of its lead candidate EPI-321, an epigenetic therapy for the neuromuscular disorder, ...

Good morning, and welcome to Sarepta's preliminary data readout of its Phase I/II studies for DM1 and FSHD. As a reminder, today's program is being recorded. At this time, I'll turn the call over to ...

Mirecule, Inc., a leader in Antibody RNA Conjugate (ARC) therapeutics targeting rare neuromuscular diseases, today announced an expansion of its strategic collaboration with Sanofi. This collaboration ...

- Potential one-time therapy for the treatment of FSHD includes a novel non-cutting dCas protein delivered via a single AAV - - EPI-321 is the only therapy designed to target the epigenetic root cause ...

Epicrispr, a clinical-stage company pioneering gene-modulating therapies, today reported early clinical data from its ongoing first-in-human, open-label study evaluating EPI-321, an investigational ...

-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and ...