FDA Paves Way for Capricor to Hold Phase 2 Trial of CAP-1002 for Duchenne MD

The U.S. Food and Drug Administration has approved Capricor Therapeutics’ plan for further development of its CAP-1002 stem cell therapy for Duchenne muscular dystrophy, paving the way for another Phase 2 clinical trial of the treatment this year. The thumbs-up came when Capricor received official minutes from a…

Muscular dystrophy (MD) is a neuromuscular disorder, characterized mainly by progressive muscle loss. Physical therapy plays an important role in MD treatment, however, there are many myths out there about the practice and its benefits. The American Physiotherapy Association has useful information to set some of these myths straight. You need…

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows. “This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to…

Olympian and fastest man on the earth, Usain Bolt, has teamed up with watch makers Hublot to design a special edition watch that will be auctioned in support of Duchenne muscular dystrophy (DMD) research, according to TMZ.com. MORE: Six complications of muscular dystrophy The 18-carat gold and sapphire watch is…

According to the Muscular Dystrophy Association of New Zealand, there are some vaccinations that people who have a neuromuscular disorder should have on an annual basis, as well as other considerations to make about vaccinations. MORE: Talking to family members about your child’s neuromuscular disorder. The current guidelines in New…

Long after it arrived elsewhere, Emflaza (deflazacort) became the first corticosteroid that the U.S. Food and Drug Administration approved to treat all forms of Duchenne muscular dystrophy — in February 2017. But its U.S. introduction was far from smooth. In fact, it took three more months and a change of…

The U.S. Food and Drug Administration (FDA) has granted Capricor Therapeutics rare pediatric disease designation for CAP-1002, its candidate therapy to treat  Duchenne muscular dystrophy (DMD). This latest approval, along with the FDA’s recent approval of orphan drug status to CAP-1002, “underscores the urgent need for treatment options for this devastating…

Muscular dystrophy is a genetic condition that causes muscle weakness and a loss of muscle mass. There is no cure for any type of muscular dystrophy but there are treatments that can help patients manage the symptoms and improve their quality of life. MORE: Watch what happens when children meet a man…

With nearly 7,000 rare diseases, including Duchenne muscular dystrophy, affecting an estimated 30 million Americans, it’s hard for Paul Melmeyer to keep track of them all. As director of federal policy at the National Organization for Rare Disorders (NORD), Melmeyer’s job is to represent the rare disease patient in…

Parent Project Muscular Dystrophy (PPMD), a nonprofit working to end Duchenne muscular dystrophy (DMD), has recognized Gillette Children’s Specialty Healthcare Neuromuscular Clinic as a Certified Duchenne Care Center. The Certified Duchenne Care Center Program was launched by PPMD in March 2014 as part of its Transforming Duchenne Care Initiative.