2109 Clinical & Scientific Conference Highlight: Healthcare Technology Moves Out of the Clinic, Into the Pocket

Computers, laptops, tablets, and smartphones have become ubiquitous features of our daily lives — but not of our healthcare. At least, not yet. But a group of physicians at MDA’s 2019 Clinical & Scientific Conference made a few convincing cases for increased use of personal electronics in healthcare and clinical trials. Dr. James Berry of . . .

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2019 Clinical & Scientific Conference Highlight: Neuromuscular Disease Impacts Cognition and Behavior, Too

Healthcare providers who work with patients diagnosed with neuromuscular disease (NMD) must consider the impact of these diseases on cognition and behavior, said presenters at a 2019 Clinical & Scientific Conference session focusing on mental health. Indeed, there is an indirect connection between NMD and mental health, but Dr. John Day from Stanford University, Dr. . . .

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2019 Clinical & Scientific Conference Highlight: Physical, Occupational, and Speech Therapy Considerations in NMD

During a late-morning session on the final day of the 2019 Clinical & Scientific Conference, five healthcare professionals — Laurie Sterling, a speech therapist at the Texas Children’s Hospital; Dr. Roxanna Bendixen, an occupational therapist and assistant professor at the University of Pittsburgh; Jennifer Chapin, a speech therapist at the University of Florida; Dr. Melissa . . .

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DMD Patients Invited to Participate in a Survey About Symptoms and Daily Life

Researchers at the University of Rochester in New York seek patients diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This study will help to determine the most critical symptoms of children, young adults, and adult patients with DMD, and as a result, . . .

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FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old

On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and 5 years old. Emflaza was approved by the FDA in February 2017 for the treatment of DMD in patients 5 years and older, making it the first drug approved . . .

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Congress is Considering a Law to Strengthen Newborn Screening: Help MDA Promote its Passage

On May 2, Reps. Lucille Roybal-Allard (CA-40), Mike Simpson (ID-02), Katherine Clark (MA-05), and Jaime Herrera-Beutler (WA-03) introduced the Newborn Screening Saves Lives Reauthorization Act of 2019, a key piece of legislation that fights to eliminate preventable newborn deaths and stave off the onset of symptoms associated with severe disabilities, including neuromuscular conditions. Read MDA’s . . .

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JAMA Neurology Publishes Article on Newborn Screening and Neuromuscular Disorders

Earlier this week, JAMA Neurology published an article co-authored by Muscular Dystrophy Association (MDA) staff members that addresses the role of newborn screening (NBS) in the field of neuromuscular disease. In the article, “Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps,” the . . .

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Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

On April 8, Audentes Therapeutics announced that it will expand its gene-targeted therapy platform to include developing experimental drugs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The company has entered into a licensing agreement with Nationwide Children’s Hospital in Ohio and will collaborate with leading researchers in the field, Kevin M. Flanigan, . . .

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Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular dystrophy (DMD). Earlier this year, the U.S. Food and Drug Administration (FDA) accepted Sarepta Therapeutics’ New Drug Application (NDA) seeking approval under Priority Review for . . .

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Buy a Shamrock, Grow Hope

It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide to raise a little green (more than $323 million, to be exact) both to send kids to MDA Summer Camp and to fund groundbreaking research . . .

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