From the time of his wife’s amyotrophic lateral sclerosis (ALS) diagnosis in 2000, Warren Schiffer and his wife, Toni Diamond, dedicated themselves to raising funds to find a cure. The couple founded Wings Over Wall Street and with the support of MDA have been able to host this annual event to raise awareness and money . . .
In 2015, Peter Warlick was diagnosed with amyotrophic lateral sclerosis (ALS). Despite the progression of the disease, Peter continues to be a dedicated father, husband, and American Airlines employee. Peter chose to make a difference for others and the future of ALS. He gathered a team, Warlick’s Warriors, and has raised more than $5 million . . .
In 2018, Sandy Morris, wife and mother of three, was diagnosed with amyotrophic lateral sclerosis (ALS) at the age of 51. Sandy is a passionate advocate for ALS research who aspires to make ALS a priority on this planet with the help of MDA and I am ALS. She has a powerful support group in . . .
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 . . .
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 . . .
With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a robust, state-of-the-art gathering for both clinicians and investigators from across the nation. Medical and scientific experts in neuromuscular disease from academia, private practice, government, and . . .