At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .
On May 6, the U.S. Food and Drug Administration (FDA) announced the approval of Ruzurgi (amifampridine) for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS) who are between 6 and 17 years of age. This is the second FDA-approved treatment for LEMS and the first approved treatment for pediatric LEMS patients. Ruzurgi is an oral potassium . . .
At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .
We are pleased to announce that on May 1, 2019, the American Society for Gene and Cell Therapy awarded MDA the Sonia Skarlatos Public Service Award for 2019. Named for its inaugural co-recipient and tireless gene therapy advocate, the ASGCT’s Sonia Skarlatos Public Service Award recognizes a person or group that has consistently fostered and . . .
In April, MDA held its first ever combined Clinical & Scientific Conference, themed “Progress in Motion,” in Orlando Fla. Clinicians, scientists, policymakers, nonprofit, and industry leaders convened for a dynamic and informative five days in Orlando, Fla. With more than 1,200 attendees, 23 sessions, 136 presentations, and 300 posters, this was MDA’s largest conference ever. . . .
Not long ago, supportive care and mobility aids were all medical science could offer people living with neuromuscular disease. But the treatment and care landscapes have begun to change dramatically. Recent breakthroughs in genetic medicine, along with other discoveries, are accelerating the development of new therapies, including some that not just address symptoms but also . . .
Yesterday, Biogen released a letter to the ALS community regarding its experimental therapy, tofersen (formerly known as BIIB067), an antisense oligonucleotide being studied for the potential treatment of amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) genetic mutation, which is a subtype of familial ALS that makes up 2 percent . . .
In a study conducted in mice, scientists led by an MDA-funded researcher found that increasing the amount of a certain mitochondrial membrane protein, mitofusin-1 (MFN1), lessened symptoms of Charcot-Marie-Tooth disease (CMT) type 2A. The findings suggest that it may be possible to treat CMT2A using gene-replacement therapies that deliver functional copies of missing or mutated . . .
Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study. The goal of the study is to characterize the long-term effects of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense mutation (which . . .
MDA’s first-ever combined Clinical & Scientific Conference kicked off today at the Hyatt Regency Orlando in Florida. MDA President & CEO Lynn O’Connor Vos and MDA Chairman of the Board Dr. R. Rodney Howell opened the event. “MDA is proud to convene this esteemed group, and we are grateful that you have joined us for . . .
