Archives




Blog Post | News, Science + Research

FDA Approves Sarepta’s ELEVIDYS for Treatment of DMD

On June 22, the US Food and Drug Administration (FDA) granted accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne…

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


Blog Post | Lifestyle

Summertime Travel Go-Anywhere Products and Tips

Packing for vacation when you live with a disability requires a little extra planning and preparation. Travel often demands additional considerations to provide easy access to essentials and ensure that…

Tags: Community, Travel


Blog Post | News, Science + Research

FDA Approves argenx’s Vyvgart Hytrulo for Treatment of gMG

On June 20, the US Food and Drug Administration (FDA) granted marketing approval of subcutaneous (SC) Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of generalized myasthenia gravis (gMG)…

Tags: Drug Approval


Blog Post | Finding a Diagnosis, Independence, Personal Stories

Hope for the Future: A Family Living with Myofibrillar Myopathy Zaspopathy Raises Awareness for Rare Disease Research

Janet Rodenhouse and her sisters have a lot in common. The seven women share a love for dancing, a deep appreciation of finding humor in all things, and a strong…

Tags: Community, Fundraising, Genetic Testing, Relationships, Research


Blog Post | Health, Personal Stories

A Letter to Muscular Dystrophy

Dear Muscular Dystrophy, MDA National Ambassador Amy Shinneman At times you dazzle me, showing me the heights of human love and kindness, and at other times you take me to…

Tags: Ambassadors, Amy, Mental Health, Relationships


Blog Post | Advocacy, Finding a Diagnosis, Personal Stories

How One Family’s Myasthenia Gravis Journey is Helping Others

Charlotte and Elizabeth Madole. For many individuals living with a rare disease, the road to diagnosis can be extremely challenging, especially when it involves two co-existing diseases where two parts…

Tags: Community, Healthcare, MDA Care Centers, Parenting, Research


Blog Post | Science + Research

Research Study Alert: Natural History Study in Males with BMD

Researchers at the University of Florida are seeking adult males living with Becker muscular dystrophy (BMD) to participate in a three-year natural history study. The objective of this observational study is to…

Tags: Research Study Alert


Blog Post | Lifestyle, News, Personal Stories

In Case You Missed It…

Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and…

Tags: Community, In Case You Missed It


Blog Post | Lifestyle, Personal Stories

MDA Ambassador Guest Blog: Happy Father’s Day

Nevin Steiner and his children, Abbott and Laurel. Being a father is one of the most rewarding things in life. I have two children, Abbott (6) and Laurel (3). Add having…

Tags: Ambassador Guest Blog, Ambassadors, Caregiving, Relationships, Staying Active


Blog Post | Get Involved, Lifestyle

Experience the Power of Community at MDA In-Person Events

As MDA welcomes back a busy schedule of in-person events this year, there are a myriad of ways that individuals can come together and reap the benefits of time well…

Tags: Community, Fundraising, Muscle Walk, Team Momentum, Volunteers, Year of the Volunteer