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Blog Post | Personal Stories, Science + Research

Do You Know ALS? Meet Sandy.

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…

Tags: Clinical Trials, Community


Blog Post | News, Science + Research

FDA Approves Jacobus Pharmaceuticals’ Ruzurgi for Treatment of Children with Lambert-Eaton Myasthenic Syndrome

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…

Tags: Clinical Trials, Drug Approval, Research


Blog Post | News, Science + Research

MDA Wins 2019 Sonia Skarlatos Public Service Award

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…

Tags: Gene Therapy, Grants


Blog Post | Personal Stories, Science + Research

Do You Know ALS? Meet Brant.

For Dane Boersma, the best things in life were simple: a good cup of coffee, and some good people to drink it with. Dane Boersma, left, with his son, Brant,…

Tags: Community, Fundraising


Blog Post | Personal Stories

MDA National Ambassador Justin Moy Reflects on Our 2019 Clinical & Scientific Conference

If I could describe the 2019 MDA Clinical & Scientific Conference in Orlando, Fla., with one word, it would be “massive” — but fortunately, I have an entire blog post…

Tags: Ambassadors, Innovation, MDA Clinical and Scientific Conference, Technology, Young Adults


Blog Post | Personal Stories

National Ambassador Tana Zwart’s Take on MDA’s 2019 Clinical & Scientific Conference

Tana Zwart When getting acquainted with something new, you can toe-test or you can jump. As for my newly appointed MDA National Ambassador role, attending the inaugural MDA Clinical &…

Tags: Ambassadors, Innovation, MDA Clinical and Scientific Conference, Research, Young Adults


Blog Post | News, Science + Research

Progress in Motion: MDA’s 2019 Clinical & Scientific Conference Heralds New Directions for Neuromuscular Disease Treatment

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…

Tags: Clinical Trials, Gene Therapy, Healthcare, Innovation, MDA Clinical and Scientific Conference, Newborn Screening, Research, Technology, Transitions


Blog Post | News

Read the New MDA Manifesto

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.…

Tags: Innovation


Blog Post | News, Science + Research

Biogen Releases Letter to ALS Community

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…

Tags: Antisense Oligonucleotide, Clinical Trials, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Mouse Study Finds Link Between Key Mitochondrial Protein and CMT2A, Making It a Possible Therapeutic Target

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…

Tags: Gene Therapy, Grants, Research