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Simply Stated: What is Spinal Muscular Atrophy?
Spinal muscular atrophy (SMA) is a neuromuscular disease that affects and impairs voluntary muscle movement. It is the leading genetic cause of infant death. SMA is caused by a mutated…
The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS
The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators…
Tags: Clinical Trials, Drug Development, Grants, Research
Scholar Rock Announces Positive Results from Phase 1 Study of SRK-015 to Treat SMA
Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July…
Tags: Clinical Trials, Drug Development, Research
Simply Stated: Is Muscular Dystrophy Genetic?
The muscular dystrophies are a group of neuromuscular diseases that cause weakness and degeneration of the skeletal (voluntary) muscles. Muscular dystrophies are generally considered genetic diseases, or diseases that arise…
Tags: Exon Skipping, Simply Stated
US House Passes Newborn Screening Reauthorization
Thanks in large part to the work of MDA advocates across the country, the US House unanimously moved yesterday to pass the Newborn Screening Saves Lives Reauthorization Act (HR 2507).…
MDA Awards Venture Philanthropy Funding to Rachelle Crosbie to Develop a Novel Membrane-Stabilizing Drug for DMD
MDA has awarded MDA Venture Philanthropy (MVP) funding totaling $389,463 over two years to Rachelle H. Crosbie, PhD, professor and chair of Integrative Biology and Physiology at the University of…
Tags: Drug Development, Grants, Research
Congress Moves Forward on Newborn Screening
Last week, the US House Committee on Energy and Commerce’s Subcommittee on Health considered the Newborn Screening Saves Lives Reauthorization Act (HR 2507), a bill that would reauthorize the national…
The Ups and Downs of Accessible Air Travel
A little knowledge and preparation can get your travel plans off the ground
Tags: Accessible Air Travel, Thrive 365, Travel
These 7 Things Can Help You Understand Gene Targeting Therapy
In recent years, drug developers have been able to pair the ever-expanding pool of gene identifications with advances in gene-targeted therapies. Researchers look to treat genetic disease at its source.
The State of Gene Targeted Therapies
How these promising therapies are being studied and tested for neuromuscular diseases
Tags: Drug Development, Featured Content, Gene Therapy, MDA Clinical and Scientific Conference, Research