Archives
Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study
Researchers at Amicus Therapeutics are looking for participants (age 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
Casey’s General Stores Round Up for MDA
This September, at Casey’s General Stores in 16 states across the Midwest, employees have been doing more than serving guests. They’ve 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
Here’s what’s new in the world of adaptive play
Tags: Featured Content
More Than a Game
A woman with CMD finds community and purpose through gaming
Tags: Community, Innovation, Mental Health, Relationships
NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping
On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA)…
Amy and Jamie Shinneman Find Purpose on Their Way to the Bank of America Chicago Marathon
For the last four months, Amy Shinneman has been training to do something she never imagined doing: hitting the pavement as a participant in the Bank of America Chicago Marathon.…
Tags: Personal Stories, Team Momentum
At a Cedars-Sinai Care Center, Dr. Robert Baloh Looks Under — and Ahead of — the Microscope for CMT and ALS
Daniel and Gladis Martinez with their daughters Genesis and Isabella Even though the Martinez family lives in Burbank, Calif., within an hour’s drive to Los Angeles’ Cedars-Sinai Medical Center, their…
Tags: Grants, MDA Care Centers, Muscle Walk, Research, Summer Camp
Five Questions with LGMD Researcher Martin Childers
Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MDA research grant totaling $192,500 over one year to perform pre-clinical studies using…
Tags: Gene Therapy, Grants, Research