Archives
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
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…
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
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
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
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
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
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
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…
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…