Archives
“How Muscular Dystrophy Changed my Life”: A Diagnosis that Led to an Incredible Cross-Country Journey
In May 2019, Jon Olson set out from Astoria, Oregon to bike across the US. He’s dedicating his miles to MDA, the research and care it supports, and the community…
Tags: Fundraising, Staying Active
Biogen Announces Positive Long-Term Results from Phase 2 Study of Spinraza to Treat SMA in Pre-Symptomatic Infants
Biogen announced positive long-term results from its ongoing phase 2 (NURTURE) clinical trial evaluating Spinraza (nusinersen) for treating spinal muscular atrophy (SMA) at the Cure SMA Annual Conference held June 28 through July 1…
This Back-to-School Season, MDA National Ambassador Justin Moy Looks Ahead to Sophomore Year
School creeps up on you! At first you look at your three-and-a-half months on the calendar and marvel at the expansive possibilities, but here I am three months later and…
Faith Goes Camping: Our National Ambassador Attends Her First MDA Summer Camp
Reagan Imhoff, 14, is a former MDA National Ambassador (2014-2015) and a current camper at MDA Summer Camp in Wisconsin. Reagan, who lives with spinal muscular atrophy (SMA), thoroughly enjoys…
Tags: Ambassadors, Summer Camp
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