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Capricor Therapeutics Announces Positive Interim Results from Phase 2 Study of CAP-1002 to Treat DMD
In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the companyβs lead investigational therapy for Duchenne muscular dystrophy (DMD),…
Tags: Clinical Trials, Drug Development, Research
MDA Grants Charles Thornton More Than $1 Million in New Funding to Continue Supporting the Myotonic Dystrophy Clinical Research Network
Charles Thornton, MD, professor of Neurology at the University of Rochester, was awarded an MDA clinical research network grant (CRNG) totaling $1,118,673 over three years to continue to lead the…
Tags: Clinical Trials, Grants, Research
Reporting Back from the MDA Public Policy and Advocacy Conference β and Keeping Up the Momentum
Two years ago, I remember looking through the photos from the MDA Public Policy and Advocacy conference, excited to see so many in the neuromuscular disease community on Capitol Hill…
Five Questions with DMD Researcher Matthew Wood
Matthew Wood, PhD, professor of Neuroscience at the University of Oxford and director of both the Oxford Rare Disease Center and the MDUK Oxford Neuromuscular Center, was awarded an MDA…
Tags: Exon Skipping, Gene Therapy, Grants, Research
ReveraGen Presents Positive Interim Results from Phase 2 Extension Study of Vamorolone to Treat DMD in Young Boys
At the World Muscle Society annual meeting held earlier this month in Copenhagen, Denmark, ReveraGen announced positive results from its ongoing phase 2 clinical trial of vamorolone for treating Duchenne…
Tags: Clinical Trials, Grants, Research
Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study
Researchers at Amicus Therapeutics are looking for participants (ageΒ 0-18) with late-onset Pompe disease (LOPD) to participate in a phase 3 study. The goal of the study is to evaluate the…
Tags: Clinical Trial Alert, Research
Caseyβs General Stores Round Up for MDA
This September, at Caseyβs General Stores in 16 states across the Midwest, employees have been doing more than serving guests. Theyβve been serving the MDA community, too, inviting store guests…
Tags: Fundraising, Summer Camp
Clinical Trial Alert: Momenta Pharmaceuticals Seeks Participants for a Phase 2 Myasthenia Gravis Study
Researchers at Momenta Pharmaceuticals are looking for adults with generalized myasthenia gravis (gMG) to participate in a phase 2 study. The goal of the study is to evaluate the safety…
Tags: Clinical Trial Alert, Research
Wheels Like Mine
The beauty and challenge of raising a child with SMA while living with SMA.
Tags: Featured Content, Parenting, Relationships
What’s the Toy Story
Hereβs whatβs new in the world of adaptive play
Tags: Featured Content