Author Archives: Margarida Azevedo, MSc

Ryan Getzlaf is hosting the 8th Annual Getzlaf Golf Shootout Sept. 8 to raise funds for CureDuchenne and its mission to find a cure for Duchenne muscular dystrophy (DMD). Getzlaf is the captain of the Anaheim Ducks of the National Hockey League. The two-day charity golf event will bring…

Sarepta Therapeutics and Invitae, a company specializing in genetic diagnostics, are expanding a partnership to help clinicians more quickly identify people with Duchenne muscular dystrophy (DMD). Sarepta is working to advance a number of potential — and an approved — disease-modifying treatments for DMD in a series of clinical…

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide Children’s Hospital. The designation allows priority review for the…

Duchene muscular dystrophy (DMD) patients now have a new scholarship program that could help them pursue their post-high school education goals. Sarepta Therapeutics recently launched “Route 79, the Duchenne Scholarship Program” to help up to 10 students who are taking the lead and making educational choices to…

The U.S. Food and Drug Administration (FDA) recently granted fast track designation to Acceleron Pharma’s ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). ACE-083 is a locally-acting agent that binds to and inhibits proteins in the transforming growth factor (TGF)-beta family, such as myostatin, which reduce…

A national survey to identify how Americans with disabilities are coping with work and overcoming barriers to employment has found that, despite persistent challenges, their efforts are making a big difference. The 2015 Kessler Foundation National Employment and Disability Survey (KFNEDS) was conducted by a multidisciplinary team of researchers at…

Parent Project Muscular Dystrophy (PPMD) has selected PerkinElmer to provide genetic testing for its Decode Duchenne program, which offers testing and counseling at no cost to eligible Duchenne muscular dystrophy (DMD) patients and their families. The DMD gene is located on the X chromosome — half of the X and…

The Parent Project Muscular Dystrophy (PPMD) is holding its 24th annual conference this year from June 27 to July 1 in Scottsdale, Arizona. Patients, families, physicians, industry partners, and other members of the community will gather at the event to discuss current issues related to Duchenne’s muscular dystrophy…

As new diagnostic tools and treatments help patients with Duchenne muscular dystrophy (DMD) live into adulthood, new challenges arise in areas like education, independence, personal relationships, health, and intimacy. To address the emerging needs of patients, caregivers and physicians of DMD patients, the Centers for Disease Control and…

Plasticity Brain Centers has become an authorized care provider for children and young adults with muscular dystrophy and other rare disorders who receive Gardiner Scholarships from the State of Florida. In addition to those with rare diseases, the scholarships allow youngsters with developmental disorders and other conditions to attend a private school or…