News

Interim results from the Phase 1/2 trial clinical trial investigating CAP-1002 as a treatment for heart disease associated with Duchenne muscular dystrophy (DMD) are being presented today at the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference taking place in Chicago. Linda Marbán, president and CEO of Capricor Therapeutics, is talking about the company’s plans…

Parent Project Muscular Dystrophy (PPMD) has added a pre-conference workshop to this year’s 23rd Annual Connect Conference, inviting international medical experts to discuss inflammation and immunity in Duchenne muscular dystrophy (DMD). The conference, set for June 29-July 2, is the largest international conference dedicated entirely to DMD. Each year, the event…

Researchers say it is important to carefully assess the timing of the start of corticosteroid treatment for Duchenne muscular dystrophy (DMD) in children and its potential association with disease outcomes. The study, “Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy,” was published in the…

Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused by nonsense mutations, including Duchenne muscular dystrophy (DMD), raising the total round of financing to $30 million. The investment will be used to advance the development of ELX-02, the…

Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent findings will…

Raxone (idebenone)  has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK). The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted Santhera Pharmaceuticals‘ drug candidate a positive Early Access to Medicines Scheme (EAMS)…

Sarepta Therpeutics and Genethon have signed a research collaboration agreement to jointly develop a gene therapy for Duchenne muscular dystrophy (DMD). Created by the AFM-Telethon, the French Muscular Dystrophy Association, Genethon is a nonprofit organization that specializes in the discovery and development of drugs for orphan genetic…

Researchers at Saint Louis University found that a nuclear receptor called REV-ERB is an important regulator of different steps in the process of muscle cell regeneration. According to the study “Rev-Erb co-regulates muscle regeneration via tethered interaction with the NF-Y cistrome,” which was featured in the journal Molecular…

The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California. The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five…

Simultaneously inhibiting three proteins can increase muscle mass, a finding that could help those with diseases such as muscular dystrophy and cancer that cause muscle deterioration, an Australian study reports. The study, “Specific targeting of TGF-β family ligands demonstrates distinct roles in the regulation of muscle mass in health and…