Category: Research

News, Research

2019 Clinical & Scientific Conference Highlight: What’s New in Neuromuscular Diseases?

Jeanene Swanson

At the 2019 MDA Clinical & Scientific Conference, one scientific session of interest centered on what’s new in neuromuscular diseases (NMDs). In this session, presenting scientists discussed progress in therapy development and clinical trials for a range of NMDs, including Becker muscular dystrophy (BMD), congenital muscular dystrophy (CMD), congenital myasthenic syndromes (CMS), myotonic dystrophy (DM), . . .

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News, Research

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

Ori Rokach

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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News, Research

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

Ori Rokach

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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News, Research, Living with Muscular Dystrophy

The Christopher Project Report to the Myotonic Dystrophy Community is Now Available

Jeanene Swanson

The results of the Christopher Project have been published in a comprehensive, 80-page report. The creation of “The Christopher Project Report to the Myotonic Dystrophy Community” was supported by several organizations, including the Muscular Dystrophy Association (MDA), and is available for download on MDA’s website. (Printed copies are also available upon request.) What is the . . .

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Research

Q&A With SSSI-MDA Fellow and Researcher Adam Bittel

Jeanene Swanson

Adam Bittel, PT, DPT, PhD, a postdoctoral fellow at Children’s National Medical Center in Washington, DC, was awarded the 2019 SSSI-MDA Fellowship Award. The award, co-sponsored by Strength, Science & Stories of Inspiration (SSSI) and MDA, will provide a total of $40,000 over two years to support Dr. Bittel’s work investigating the cellular mechanisms underlying the effects . . .

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Personal Stories, Adults, Research, Living with Muscular Dystrophy

Researcher with FSHD Awarded MDA Funding to Discover New Therapies for the Disease

Jeanene Swanson

Like many pre-medical students, Justin Cohen discovered along the way that what he really liked was research. However, unlike others who exchange the stethoscope for a microscope, Justin had a strikingly different motivating factor — he has been living with the disease he studies, facioscapulohumeral muscular dystrophy (FSHD), for almost as long as he can . . .

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News, Research

MDA Awards Venture Philanthropy Funding to Locana to Develop Novel Treatment for DM

Jeanene Swanson

Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy (DM), the most common form of adult-onset muscular dystrophy. MVP is the MDA’s drug development program that is exclusively focused on funding the discovery and . . .

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Kids, Young Adults, Adults, News, Research, Living with Muscular Dystrophy

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

MDA Staff

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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Kids, News, Research

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

Jeanene Swanson

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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Adults, Research

Do You Know ALS? Meet Dr. Cudkowicz.

MDA Staff

Dr. Merit Cudkowicz, chief of Neurology at Massachusetts General Hospital, where the ALS Multidisciplinary Clinic in the Healey Center for ALS is associated with MDA, is dedicated to performing academic-led clinical trials in amyotrophic lateral sclerosis (ALS). Her internationally renowned research has led to advanced treatments for people with living with ALS. What role has . . .

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