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Treatment for FSHD named orphan drug, put on FDA fast track
The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body. The experimental muscle-targeted therapy, which aims to improve motor function in FSHD, is…
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Guest Voice: 10 years after my son’s desperate plea to the FDA
On April 25, 2016, my son Billy stood at a podium in a hotel ballroom outside Washington, D.C., and told a room full of advisers from the U.S. Food and Drug Administration (FDA) something no 15-year-old should have to say out loud. “FDA, please don’t let me die early,” he said. This September marks 10…
Support Our Endeavor to Expedite the
Discovery of a Cure
Enlist your support or make a contribution today to advance our mission
Helping Those in Need
Our Commitment
Our Mission
Our mission is to raise funds for the best global research efforts to find cures for every type of Muscular Dystrophy and to put an end to the terrible suffering of hundreds of thousands of children. We collect as much data as possible on different children with different types of muscular dystrophy and share the data with doctors and scientists around the world to expedite finding and delivering a cure.
Our Goal
Our goal is to cure every type of muscular dystrophy by funding cutting edge technologies around the world, and to accelerate the processes of finding the cure through the collective knowledge of scientists, doctors and other professionals that are willing to join our cause. In order to find a cure quickly without being hampered by political and big-corporation red tape, we aim to raise funds, knowledge, and human capital on a global scale.
Marching for a Cure: Our Anthem of Hope.
Who We Are
Our Story
We are a small dedicated group with the sole goal of curing muscular dystrophy (MD). We are working on…
Our Team
Team DMD Warriors unites diverse talents in a relentless pursuit of a cure for Duchenne Muscular Dystrophy…
Our Friends
Our circle of friends stands united in the fight against Duchenne Muscular Dystrophy. Fueled by love and a shared commitment…
Supporting Children
Helping Kids
Helping children with Duchenne muscular dystrophy (DMD) involves a multidisciplinary approach that addresses both their physical and emotional well-being.
This may include physical therapy to maintain mobility, adaptive devices to enhance independence, and emotional support for both the child and their family as they navigate the challenges associated with this progressive muscle-wasting condition. Staying informed about the latest medical advancements.
Our Quest is Centered on Unraveling the Complexities of DMD Treatment
Our Projects and Goals
We concentrate our efforts in funding research in four main areas.
- Utrophin Upregulation
- Exon Skipping
- Gene Therapy: Micro-Dystrophin 4500 bases
- Gene Therapy: AAV.Mini-Dystrophin Overlapping Vectors 6400 bases
Finding a Cure
Research and Hospitals
Medical Research
Guiding Humanity Towards a Better Tomorrow
Shriners
Change and Improve Lives
Locations
22 hospitals
Medical Research
DMD research drives transformative breakthroughs, offering hope to those affected. Researchers worldwide collaborate to reshape the medical landscape, striving to improve lives impacted by Duchenne Muscular Dystrophy.
About Shriners
Shriners Children’s Hospital is a beacon of specialized pediatric care and other complex medical needs. Committed to providing exceptional care, the hospital emphasizes family-centered support and is source of hope for children worldwide.
Shriners Locations
Shriners Hospitals for Children oversees a network of 22 hospitals spanning North America. To assist you in locating the nearest facility for you and your family, the link below containing a list of hospitals along with their contact information.
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