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Blog Post | Health, Science + Research

Announcing the 2024 MDA Clinical & Scientific Conference Agenda

The 2024 MDA Clinical & Scientific Conference is just around the corner. We’re looking forward to you joining us, as we showcase groundbreaking research and clinical accomplishments by dedicated professionals…

Tags: Gene Therapy, Healthcare, Innovation, MDA Clinical and Scientific Conference, Research


Blog Post | Science + Research

9 Questions to Ask Before Joining a Clinical Trial

Experts recommend asking these nine questions before joining a clinical trial so you know what to expect.

Tags: Clinical Trials, Featured Content, Gene Therapy, MDA Care Centers, Research


Blog Post | Advocacy, Get Involved, Health, Inclusion, Independence, Lifestyle, Personal Stories

Episode 36- Wrapping Up 2023 with Leah and Amy

In this Quest Podcast episode, we chat with Muscular Dystrophy Association’s National Ambassadors, Leah Z., and Amy Shinneman. Amy feels that connecting and sharing her story with others through MDA…

Tags: Ambassadors, Amy, Community, Genetic Testing, Healthcare, Leah, Mental Health, Relationships, Staying Active, Young Adults


Blog Post | Finding a Diagnosis, Health, Science + Research

Simply Stated: Research Updates in Leigh Syndrome

Leigh syndrome is a rare, neurodegenerative disorder caused by defects in the mitochondria, the energy-producing structures within our cells. This disorder often begins in infancy or early childhood and causes…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development, Education, Healthcare, Innovation, MDA Care Centers, MDA Resource Center, Research, Resources, Simply Stated


Blog Post | Finding a Diagnosis, Health

6 Questions to Ask Your Doctor to Get a Diagnosis

When doctors don’t know what you have, ask your healthcare providers these important questions to help them get to a diagnosis.

Tags: Featured Content, Healthcare


Featured Article | Health, Independence

Make a Smooth Transition From Pediatric to Adult Healthcare

What you need to know to make a smooth transition from pediatric to adult healthcare for muscular dystrophy and other neuromuscular diseases.

Tags: Featured Content, Healthcare, MDA Care Centers, Transitions, Young Adults


Blog Post | Advocacy, Get Involved, Inclusion, Independence, Lifestyle, Personal Stories

Episode 35- Bridging the Gap Between Intention, Action, and Community Inclusion

In this Quest Podcast episode, we chat with the Director of Marketing and Communications at Idealist.org, President for New York Women in Communications (NYWICI) and host of the NYWICI WomenHeard Changemakers podcast.…

Tags: Community, Mental Health, Relationships, Resources, Staying Active, Volunteers


Blog Post | Health, News, Science + Research

AGAMREE ® (Vamorolone), Now FDA-Approved, Offers Hope for Children Living with Duchenne Muscular Dystrophy

The recent FDA approval of Catalyst’s AGAMREE ® (vamorolone) is a beacon of hope for people living with Duchenne muscular dystrophy (DMD), a rare but serious degenerative muscle disease.   According…

Tags: Drug Approval, Drug Development, Healthcare, Innovation, Research Advances


Blog Post | Health, Lifestyle

Coping with Family Caregiver Fatigue

Tips for family caregivers to recognize the signs of caregiver burnout and manage stress to prevent it.

Tags: Caregiving, Community, Featured Content, Mental Health, Relationships


Blog Post | Health, News

Insurance Denials for Gene Therapy Treatment Delay Access to Care

The FDA approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), the first-ever, gene therapy treatment for young boys living with Duchenne muscular dystrophy (DMD), brought unprecedented hope to families living with DMD. Designed…

Tags: Gene Therapy, Healthcare, Insurance, MDA Care Centers, MDA Resource Center, My Gene Therapy Journey, Resources