At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
The question of whether to go back to school amidst Covid-19 has been a debate across the country. Many universities have seen surges in cases, many involving students in Greek…
According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
CureDuchenne Webinar: Update on the Italfarmaco Givinostat Program September 24, 2020 Hear from Dr. Paolo Bettica about the latest news related to Italfarmaco's investigational therapy givinostat, which is being tested…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
For twenty-five years Women of Influence has focused on empowering and helping to advance women in the workforce. Maayan Ziv, a Canadian tech entrepreneur, disability advocate, and voted one…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…
By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
June marks the start of Aphasia Awareness Month, designed to educate many on aphasia: causes, long-term affects, and the lives of those living with this condition. But, as the University…
According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…
Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…
Parent Project Muscular Dystrophy is a nonprofit that aims to find a cure for Duchenne muscular dystrophy. One of their programs, the Certified Duchenne Care Center Program (CDCCP), has recently…
Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…
With 4.15 million cases across the globe, COVID-19 is at the forefront of discussion. Knowledge about the viral pandemic continues to grow each and every day. Yet there are still…
According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
A recent editorial focusing on NMDs (neuromuscular disorders) was published in the journal Disease Models & Mechanisms. The editorial highlights a special issue of the journal devoted to building…
Alyla Annac When Alyla Annac was just 19 years old her father passed away from heart failure. He had suffered 3 heart attacks and doctors said there was nothing else…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
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