Margarida Azevedo, MSc,  —

Parent Project Muscular Dystrophy (PPMD) is promoting a series of running events this year to raise money for research to help end Duchenne muscular dystrophy (DMD). The next Run For Our Sons event will be an official charity partner of the Rock n’ Roll Raleigh Marathon, Half…

Researchers have received $119,999 in funding to test the effectiveness of a gene therapy approach in Charcot-Marie-Tooth neuropathy X type 1 (CMTX1), the second most common form of Charcot-Marie-Tooth disease (CMT). The award was granted by the Muscular Dystrophy Association (MDA) and the Charcot-Marie-Tooth Association (CMTA) to Kleopas…

A Napa Valley fundraiser in Newport Beach, California, earlier this month attracted more than 400 people and raised at least $1 million for Duchenne muscular dystrophy (DMD) research. The Napa in Newport wine auction on March 4 supported the mission of the nonprofit group CureDuchenne — to improve the lives…

Catabasis Pharmaceuticals will present results from Part B of the MoveDMD trial of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD). According to a press release, the presentation, “MoveDMD: Phase 1/2 trial of Edasalonexent, an NF-kB Inhibitor, in 4- to 7-Year-Old Patients with Duchenne Muscular…

The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening diseases. The investment reflects the nonprofit organization’s plan to double spending on drug development and clinical trials by 2020. Each year,…

The U.S. Food and Drug Administration (FDA) acknowledged the filing over protest of PTC Therapeutics’ new drug application (NDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The FDA granted Translarna standard review and assigned a target date of Oct. 24, 2017…

Tens of thousands of businesses across the United States are taking part in this year’s 35th annual Muscular Dystrophy Association (MDA) Shamrocks campaign, which aims to raise funds for research and support programs to help people with muscular dystrophy. The MDA Shamrocks campaign is the country’s largest St. Patrick’s Day fundraising…

ATyr Pharma’s Resolaris has received orphan drug status in the United States for the treatment of limb girdle muscular dystrophy (LGMD). The Food and Drug Administration designation gives companies financial and other incentives to develop therapies for diagnosing or treating rare diseases. In the United States that means fewer…

CureDuchenne Ventures will give Exonics Therapeutics $5 million to advance research on a gene editing technology known as CRISPR/Cas9 that is aimed at correcting most of the mutations leading to Duchenne muscular dystrophy (DMD). DMD is a rare X-linked genetic progressive muscle disease affecting nearly 15,000 boys in the United…

Catabasis Pharmaceuticals reported mixed topline data from Part B of the MoveDMD trial evaluating the safety and efficacy of CAT-1004 (edasalonexent) in treating Duchenne muscular dystrophy (DMD) patients. MoveDMD is a three-part, multisite Phase 1/2 trial (NCT02439216) evaluating the efficacy, safety, and the pharmacodynamics (PD) and pharmacokinetics (PK),…