Author Archives: Patricia Inacio PhD

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to progress into late-stage heart failure, similar to humans — of people with Duchenne muscular dystrophy (DMD), a study reports. Moreover, using the new mouse model, researchers gathered promising data…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

The Muscular Dystrophy Association (MDA) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate from Cambridge, Massachusetts, as its latest National Ambassador. “I have been involved with MDA because muscular dystrophy is now a part of me,” Greaves said in an MDA press release. “I…

The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for limb-girdle muscular dystrophy type 2i (LGMD2i). BBP-418 is a small molecule that delivers a prodrug — a precursor molecule — which, once inside the body, will change into the sugar…

Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD), the company announced. The company has reached an agreement with ReveraGen Biopharma, the original developer of vamorolone, and Actelion Pharmaceuticals’ spin-off Idorsia, which had acquired an…

This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually for the first time to ensure maximum participation, while promoting social distancing and other safety measures for people with…

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company announced. In a letter to Solid, the FDA has requested more information about the gene therapy’s…

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the disease, top-line data from the HOPE-2 clinical trial show. “I am incredibly pleased with the outcome of the…