Archives
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
MDA Awards Clinical Research Network Grant to CMT Researcher Michael Shy for Inherited Neuropathies Consortium
Michael Shy, PhD, professor of Neurology and Pediatrics at the University of Iowa, was awarded an MDA Clinical Research Network Grant (CRNG) totaling $423,413 over three years to further develop…
In a Letter to the Duchenne Community, Santhera Announces Positive Results of Phase 2 Vamorolone Study, EMA’s Review of Puldysa to Treat Symptoms of DMD
This week, Santhera released a letter to the Duchenne muscular dystrophy (DMD) community regarding an update on Santhera’s DMD drug development programs and its partnership with ReveraGen. The full letter…
Tags: Clinical Trials, Research, Research Advances
Five Questions with Friedreich’s Ataxia Researcher Sanjay Bidichandani
Sanjay Bidichandani, MBBS, PhD, professor of Pediatrics at the University of Oklahoma Health Sciences Center, has an ongoing MDA research grant (totaling $300,000 over three years) to study the role…
Clinical Trial Alert: Italfarmaco Seeks Participants for a Phase 3 DMD Study
Researchers at Italfarmaco are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy and…
Tags: Clinical Trials, Research
Researcher with CMT Awarded MDA Funding to Discover Rare Variants for Multiple Neuromuscular Diseases
Receiving a diagnosis of a neuromuscular disease is often not easy — there can be denial, anger, grief, and a frustrating sense that maybe life is, in a way, over.…