Author Archives: Marisa Wexler MS

#AANAM – Investigational DM1 Therapy Shows Promise

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. An investigational therapy called AOC-1001 decreased levels of the toxic RNA molecule that causes…

FDA Places Pamrevlumab for DMD on Fast Track

The U.S. Food and Drug Administration has granted fast track designation to pamrevlumab, an investigational treatment for Duchenne muscular dystrophy (DMD). The designation is given to medications that have the potential to treat serious conditions and to fill an unmet medical need. The designation allows the therapy’s developer, FibroGen,…

MDA Engage Offers Free Educational Events

Through a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational events for people affected by muscular dystrophy and other neuromuscular diseases. The events are aimed at people living with these conditions, as well as their loved ones. MDA Engage offers three types…

STRIVE Applications for Duchenne Community Now Open

Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program for Duchenne muscular dystrophy. The annual program, facilitated by PTC Therapeutics, was designed to help support initiatives that address unmet needs in the Duchenne community. Goals of STRIVE range from…

Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a small clinical trial, according to a press release. The Phase 1/2 clinical trial (NCT02667483) tested DS-5141 for a first time in Duchenne patients amenable to exon 45 skipping. No…

Pivotal Trial Begins for AMO-02 in Steinert Disease

AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02 (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), also known as Steinert disease. The double-blind trial (NCT03692312) is…

Translarna Approved in Russia for DMD Patients 2 and Older

Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages 2 and older. “Translarna was the first therapeutic ever approved for nonsense mutation Duchenne muscular dystrophy patients and we are excited to expand approval of Translarna into Russia,” Stuart…