Margarida Azevedo, MSc,  —

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Resolaris as a potential treatment for limb girdle muscular dystrophy 2B (LGMD2B). The FDA also removed its partial clinical hold on a dosing ceiling for Resolaris in clinical trials, the drug’s developer, aTyr Pharma, announced in press…

A researcher at the University of Texas Southwestern Medical Center has been awarded a $250,000 grant to  study CRISPR/Cas9 technology‘s potential to treat Duchenne muscular dystrophy (DMD). CRISPR/Cas9 allows researchers to add, remove, or change sections of the DNA sequence. Parent Project Muscular Dystrophy (PPMD) will give the…

Yale New Haven Children’s Hospital’s (YNHCH) Muscular Dystrophy Program has been named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to ending Duchenne muscular dystrophy (DMD). PPMD’s Certified Duchenne Care Center Program, which was started in 2014, supports standardized, comprehensive…

The New Jersey Institute of Technology (NJIT) and Talem Technologies will explore the potential of robotic technology to assist people living with Duchenne muscular dystrophy (DMD), using a $600,000 grant awarded by Parent Project Muscular Dystrophy (PPMD). The two-year project is titled “Investigation of the Community Use…

The European Commission has granted Orphan Drug Designation to Benitec Biopharma’s BB-301, an investigational treatment for oculopharyngeal muscular dystrophy (OPMD). The decision represents a major advance for OPMD, a rare congenital myopathy, for which the currently used therapeutic strategies involve repetitive surgical interventions that have limited effectiveness. Orphan Drug…

CureDuchenne Cares, a community-based program providing educational and outreach workshops for parents and others caring for people with Duchenne muscular dystrophy (DMD), has just announced its planned events for 2017. CureDuchenne, the non-profit DMD research support organization, pioneered the Cares program to address a perceived urgent need to educate DMD families…

Many patients with myotonic dystrophy type 1 (DM1) have sleep and respiratory disorders and may benefit from targeted therapy. New research has found that the causes of sleepiness in patients with DM1 are variable, but include poor sleep patterns, obstructive sleep apnea, respiratory failure, and narcolepsy. DM1 is the most common…

The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients. PTC Therapeutics can now market Translarna for nmDMD patients who are 5 or older, mobile, and living in the European Union, Iceland, Liechtenstein, and Norway. The decision follows a…

Researchers found notable differences in the access to appropriate care for Duchenne’s muscular dystrophy (DMD) patients in seven European countries. In a survey known as CARE-NMD, patients in DMD registries in the countries —  Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland and the United Kingdom — were questioned as to care…

Children with special healthcare needs, such as those with muscular dystrophy (MD), benefit from at-home care, but at high costs to their families. In fact, uncompensated medical care in the U.S. costs theses families up to $36 billion annually, a new comprehensive national study reported. The study, published in…