Archives
2019 Clinical & Scientific Conference Highlight: Neuromuscular Disease Impacts Cognition and Behavior, Too
Healthcare providers who work with patients diagnosed with neuromuscular disease (NMD) must consider the impact of these diseases on cognition and behavior, said presenters at a 2019 Clinical & Scientific…
2019 Clinical & Scientific Conference Highlight: Physical, Occupational, and Speech Therapy Considerations in NMD
During a late-morning session on the final day of the 2019 Clinical & Scientific Conference, five healthcare professionals — Laurie Sterling, a speech therapist at the Texas Children’s Hospital; Dr.…
The Christopher Project Report to the Myotonic Dystrophy Community is Now Available
The results of the Christopher Project have been published in a comprehensive, 80-page report. The creation of “The Christopher Project Report to the Myotonic Dystrophy Community” was supported by several…
Tags: Research
Simply Stated: What is Myasthenia Gravis?
Myasthenia gravis (MG) is an autoimmune disease, which is a disease that occurs when the immune system goes awry and produces antibodies that attack the body’s own tissues. Normally, antibodies…
Tags: Drug Approval, Research, Simply Stated
Q&A With SSSI-MDA Fellow and Researcher Adam Bittel
Adam Bittel, PT, DPT, PhD, a postdoctoral fellow at Children’s National Medical Center in Washington, DC, was awarded the 2019 SSSI-MDA Fellowship Award. The award, co-sponsored by Strength, Science & Stories of…
Tags: Grants, Research, Staying Active
10 Facts About FSHD — and a Reason to Smile
An older gentleman came up to me once. I had just been on TV for the Jerry Lewis MDA Labor Day Telethon talking about how facioscalpulohumeral muscular dystrophy (FSHD) affects…
Tags: Ambassadors, Community, Genetic Testing
Researcher with FSHD Awarded MDA Funding to Discover New Therapies for the Disease
Dr. Justin Cohen Like many pre-medical students, Justin Cohen discovered along the way that what he really liked was research. However, unlike others who exchange the stethoscope for a microscope,…
MDA Awards Venture Philanthropy Funding to Locana to Develop Novel Treatment for DM
Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy…
Tags: Drug Development, Gene Therapy, Grants, Research
FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old
On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and…
Tags: Drug Approval, Newborn Screening, Research
Congress is Considering a Law to Strengthen Newborn Screening: Help MDA Promote its Passage
On May 2, Reps. Lucille Roybal-Allard (CA-40), Mike Simpson (ID-02), Katherine Clark (MA-05), and Jaime Herrera-Beutler (WA-03) introduced the Newborn Screening Saves Lives Reauthorization Act of 2019, a key piece…