Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD

Researchers at NS Pharma are seeking boys with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping therapy to participate in a phase 2 clinical study (clinicaltrials.gov ID: NCT05996003) of their investigational therapy brogidirsen (NS-089/NCNP-02-201). DMD is caused by gene mutations that lead to the loss of the dystrophin protein, which is important for the structure…

MDA Advocacy Team Reports Updates in Newborn Screening Legislation

This year has been an eventful year for newborn screening in neuromuscular disease, with potential further progress on the near horizon. We began 2025 with all 50 states screening for spinal muscular atrophy (SMA), meaning nearly all babies born in the United States can be screened for SMA leading to a rapid diagnosis and near…

Head Back-to-School in Style with Adaptive Fashion

For many families with kids, the end of summer includes preparations for heading back to school for a new year of growth, learning, and activities. On top of school supplies, schedules, and meeting new teachers and classmates – this time of year also usually means back-to-school clothes shopping. As the fashion industry continues to make…

Clinical Research Alert: Phase 1/2 Study of SGT-003 in Boys with DMD

Researchers at Solid Biosciences are seeking boys with Duchenne muscular dystrophy (DMD) to participate in a phase 1/2 clinical trial (INSPIRE DUCHENNE) to evaluate the safety and efficacy of the investigational gene therapy (SGT-003). DMD is caused by the loss of the dystrophin protein, which is important for the structure and function of muscle cells. SGT-003…

Five Common Corticosteroid Questions and Considerations

Find answers to the five most common corticosteroid questions as well as other considerations if you’re prescribed them to treat a neuromuscular disease.

MDA Ambassador Guest Blog: Someone Like Me

My name is Santana Gums and I have LGMD-2b. I live in Arizona and work as a Legal Assistant. Back in February of this year, I attended the Arizona Muscle walk, which just so happened to be my first ever MDA event. Being new to the community, I wasn’t sure what to expect, who would…

Clinical Research Alert: Observational Study on Parent and Caregiver Perspectives on Adherence to DMD Care

Researchers at Albany Medical College are seeking parents and caregivers of children with Duchenne muscular dystrophy (DMD) to participate in an observational REDCap survey about their adherence to DMD care guidelines and challenges and priorities in managing their child’s care in the United States. The findings of this survey will help to better understand the experiences…

Simply Stated: Updates in Charcot-Marie-Tooth Disease (CMT)

Charcot-Marie-Tooth disease (CMT) encompasses a group of inherited disorders that affect movement and sensation in the arms, legs, hands, and feet. Common symptoms include lower leg weakness, foot deformities, reduced sensations, and sometimes impaired fine motor skills. While CMT typically progresses slowly, symptoms may spread over time and lead to secondary complications, such as scoliosis.…

Clinical Research Alert: Natural History Study of Individuals with Non-Dystrophic Myotonias

Researchers at the Center for Health + Technology at the University of Rochester are seeking individuals with non-dystrophic myotonias (NDM) to participate in an observational interview. The findings of the interviews will help to better understand the symptoms experienced by people with NDM. The study This observational study consists of a one-time Zoom interview that will…

National Read a Book Day: Spotlight on Community Authors

While every day is a good day to enjoy the simple pleasure of reading, National Read a Book Day on September 6th offers a fun reminder for booklovers to slow down, settle in, and enjoy a good book. This year, we want to spotlight some of the talented voices resonating within the neuromuscular disease community.…

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QUEST PODCAST

The Quest podcast, proudly presented by the Muscular Dystrophy Association, is part of the Quest family of content. Hosted by Quest Editor-in-Chief, motivational speaker and writer Mindy Henderson.

Episode 55- Unpacking Disability Pride: Voices from the MDA Community

In this Quest Podcast episode, we chat with MDA Ambassadors, Payton Rule, Fred Graves and former MDA National Ambassador Amy Shinneman. Payton shares a journey of transformation from self-doubt to pride, emphasizing how important community has been in helping her feel seen and valued.  While Fred offers a perspective rooted in resilience and advocacy, discussing…

Episode 54- Service Dogs, Inc. – Paws with a Purpose

In this Quest Podcast episode, we chat with a former attorney who left her law practice to devote her time to building Service Dogs, Inc. Sheri Soltes founded Service Dogs, Inc. in 1988 on the concept of using dogs rescued from animal shelters. Under her guidance, Service Dogs, Inc. has led the industry in combining…

Episode 53- Invisible People: Making the Rare Seen

In this Quest Podcast episode, we chat with a former pharmacist turned singer/songwriter who lives with Generalized Myasthenia Gravis. Dania Quill has devoted her time and expertise to create inclusive spaces for those with disabilities and deliver advice, inspire action, and make us feel closer through song while sharing stories of resilience and positivity.  While…