Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…
Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…
There is a growing consensus that patients need to be more included in the medical field, reports PCMA Convene. Over the last ten years increasing numbers of organisations have moved…
According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…
Clinical trials of a potential gene therapy treatment for Duchenne muscular dystrophy (DMD) have been put on hold after concerns emerged about the safety of the procedure, reports Genetic Engineering…
According to a story from Business Wire, there was a time when having to use a mechanical ventilator in order to breathe meant being confined at the hospital in an…
According to a story from Medical Xpress, two new treatments were able to slow down the worsening of symptoms associated with muscular dystrophy in a mouse model study. Tamoxifen and…
According to a story from aninews.in, mother Shashi Mishra and her daughter Anamika Mishra have staged a hunger strike in a desperate move to receive treatment for their muscular dystrophy.…
According to a story from EurekAlert!, scientists from UCLA have developed a new method of delivery for gene therapies called nanospears. These structures could allow for more accurate and precise…
CRISPR gene editing technology is still mostly in its infancy, but the new tool is already causing a lot of excitement, particularly in the treatment of genetically-linked diseases. A recent…
Muscular Dystrophy Association will host the 2018 Clinical Conference in Arlington, VA. The meeting will bring together scientific and medical experts from private practices, academia, industry and government. It will…
The Imperial College of London recently began development of a body suit to improve treatment of Duchenne muscular dystrophy. The suit uses a unique artificial intelligence interface to collect and…
Sailormen, Inc. represents one of the largest franchise groups of Popeyes Louisiana Kitchen restaurants in the United States. The company includes 136 stores across six states. How does this related…
Originally reported by Columbia Daily Tribune, researchers from the University of Missouri have made huge strides towards treating, and maybe even curing, two fatal diseases: muscular dystrophy and Amyotrophic Lateral…
One of the medical research field's biggest developments recently - within the last 5 years! - is a new form of gene editing, also know as genome editing. Gene editing refers to…
For those people struggling with GNE myopathy, there is someone who wants to hear from you. Whether you’re a patient or a loved one of someone with this rare disease,…
People find love in endless different ways. Some people meet their romantic partner through dating apps and singles groups, others meet at work or social gatherings. One place you don't…
What can one little boy and his family do to fight a rapidly debilitating and (currently) incurable disease such as Duchenne muscular dystrophy? A lot. The diagnosis of a loved…
Duchenne muscular dystrophy is most common in males, and is characterized by muscle weakness that progresses over time. Onset is as early as three years of age and the life…
Sad news today as the world mourns comedian and Muscular Dystrophy Advocate Jerry Lewis' death. The director/producer died at the age of 91. Jerry Lewis was a 1960's movie star…
If you've ever been curious about muscular dystrophy, now is the time to learn because August is Muscular Dystrophy Awareness Month! Chances are, even if you think you have no…
2017 Muscular Dystrophy Association Scientific Conference was a beacon of light! Researchers from academic medical centers, pharmaceutical companies, children’s’ medical centers, biotech, engineering, and veterinary medicine came together to share…
The Muscular Dystrophy Association's Scientific Conference Registration closes February 28th! This event brings all the important players together in the Muscular Dystrophy world to promote research around management, treatment and…
If you, or someone you love, has Duchenne muscular dystrophy (DMD), then each new possible drug that's brought before the FDA brings with it a sense of hope. Race to…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
