Stockhead on MSN3d
Health Check: Percheron ‘closes the book’ on failed childhood rare disease program
Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.
Dyne Therapeutics to Present at Oppenheimer 35th Annual Healthcare Life Sciences Conference
A live webcast will be available in the Investors & Media section of Dyne’s website at https://investors.dyne-tx.com/news-and-events/events-and-presentations and a replay will be accessible for 90 ...
JMP Securities maintains Solid Biosciences stock with $15 target
JMP Securities reiterated their Market Outperform rating on Solid Biosciences (NASDAQ:SLDB) with a steady price target of $15.00. Currently trading at $3.04, the company has a market capitalization of ...
Muscular Dystrophy News5d
Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UK
Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.
GEN6d
AAV Delivered NanoCas CRISPR System Edits Muscle in Non-Human Primates
Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.
the-tartan6d
New study on personalized medicine could speed up treatments for rare diseases
Personalized organoids — ‘mini-organs’ — may accelerate treatment for patients with rare genetic disorders. Courtesy of Europeana via Unsplash Across the globe, countless babies ...
The EMBO Journal6d
Type-2 innate signals are dispensable for skeletal muscle regeneration and pathology linked to Duchenne muscular dystrophy
Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...
The American Journal of Managed Care2d
Ifetroban Improves Heart Function in DMD
There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...