On Oct. 3, Genentech announced interim results from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). Risdiplam is an investigational SMN2-splicing modifier that is given by mouth (or g-tube) and distributes widely throughout the body. Risdiplam is designed to help the SMN2 gene produce more . . .
Researchers at Capricor Therapeutics are looking for ambulatory and non-ambulatory children and adults with Duchenne muscular dystrophy (DMD) to participate in a Phase 2 study. The goal of the study is to evaluate the safety and efficacy of CAP-1002. Researchers hope that this therapy may slow disease progression in heart and skeletal muscle by modulating . . .
On Labor Day weekend, Harley-Davidson and the Muscular Dystrophy Association hit the streets of Milwaukee to celebrate Harley’s 115th anniversary — and the 38-year partnership between the iconic American motorcycle maker and MDA. Thousands of riders and bikes convened for Harley’s celebratory 115th Anniversary Parade on Sept. 2. Within the parade was MDA’s Parade of . . .
Sept. 30th is Limb-Girdle Muscular Dystrophy Awareness Day, which celebrates individuals living with the more than 30 different subtypes of Limb-girdle muscular dystrophy (LGMD). MDA has a long-standing commitment to LGMD — we’ve invested more than $59 million in LGMD-specific research since 1950, including more than $2.8 million in current active grants. MDA Scientific Portfolio . . .
Every year on Sept. 30, people around the world wear lime green and come together online and in their communities to celebrate Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day. Now four years old, LGMD Awareness Day has been embraced by the LGMD patient community as a way to raise awareness and honor individuals living with the . . .
At 22, when I graduated from college, I, like so many of my fellow millennials, moved back home. I spent two transitional years in the room where I’d grown up, unicorn wallpaper still casting magic above the bed, and then, one weekend, I packed my clothes and moved out. I’d lived alone in my college . . .
Since 1897, the Boston Marathon has held a special place in the hearts of Bostonians. Held every third Monday in April, the Boston Marathon is one of the most famous races in the world, attracting runners of all abilities to a beautiful, grueling course that begins in the hilly western suburbs of Boston and ends . . .
In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a . . .
On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .
In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT. The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, . . .