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FDA’s Approval of Duvyzat (givinostat) Offers New Hope to People Living with Duchenne Muscular Dystrophy
On March 21, the U.S. Food and Drug Administration gave final approval to Duvyzat (givinostat), a new treatment for children and adolescents living with Duchenne muscular dystrophy (DMD). In clinical…
Tags: Clinical Trials, Drug Approval, Drug Development, MDA Care Centers, Research, Research Advances
At MDA Conference Neuromuscular Disease Experts Discuss Readiness for Gene Therapies
Are hospitals and healthcare institutions ready to administer new gene therapies? Experts weigh in at the MDA Clinical & Scientific Conference.
Tags: Featured Content, Gene Therapy, Healthcare, MDA Care Centers, MDA Clinical and Scientific Conference
MDA Ambassador Guest Blog: My Experience Participating in a Research Study
Lacey Woods is an MDA Ambassador living with LGMD2i (R9). She is a wife and mother of six children. She works with her husband in real estate and as a…
Tags: Ambassador Guest Blog, Ambassadors
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: Ambassadors, Community, Drug Development, Healthcare, In Case You Missed It, Parenting, Relationships, Resources, Summer Camp
Simply Stated: The Creatine Kinase Test
Almost everyone with a neuromuscular disease has had, or will have, a creatine kinase test. But what exactly is creatine kinase (CK), and why are its levels measured in neuromuscular…
Tags: Simply Stated
Heart of the Muscular Dystrophy Association Mission Was in Focus at the 2024 MDA Clinical & Scientific Conference Opening Day
Brooke Eby spoke to researchers at the first day of the 2024 MDA Clinical & Scientific Conference and other highlights.
MDA Announces College Scholarship Program
The Muscular Dystrophy Association (MDA) is excited to announce the launch of its inaugural MDA College Scholarship program. Continuing MDA’s endeavors to support and empower individuals living with neuromuscular disease…
Tags: College, Community, Education, Finance, MDA Q and A
Inclusion Outshines Ableism on the Big Stage
Most Likely Not To… will debut on Rare Disease Day, February 29, 2024. The disability community is making history this year with a first-of-its-kind musical production created from start to…
Tags: Accessible Air Travel, Community, Travel
Simply Stated: Muscle Biopsy
A muscle biopsy is a surgical procedure in which one or more small pieces of muscle tissue are removed for microscopic examination and biochemical or genetic testing. The procedure, sometimes…
All 50 States and DC Implement Spinal Muscular Atrophy Newborn Screening
2024 has already brought several exciting developments throughout MDA’s advocacy priorities and policy areas, but one of the biggest victories for the MDA community so far this year is the…