An expression of the live unlimited spirit, stories and voices of the MDA community

UC Davis Researchers Seek DMD Participants for Survey to Improve Clinical Trial Outcome Measurements

In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a . . .

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FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS

On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .

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Three Major Patient Advocacy Groups Focused on CMT Research to Collaborate on FDA “Voice of the Patient Report” and Patient-Focused Meeting

In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT. The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, . . .

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Exploring the Latest in MDA’s CMT Research

The future of Charcot-Marie-Tooth research and treatments rests where the disease begins: in genes. While many new genes involved in CMT have been identified, we know as many as 40 percent of people living with CMT still don’t have a confirmed genetic diagnosis. We’re also still trying to understand the disease mechanisms of the genes . . .

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