Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…
The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
Rare Community Profiles Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…
For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
Since its founding 20 years ago, CureDuchenne has been tirelessly working to find and fund a cure for Duchenne muscular dystrophy (DMD). They have done this through supporting families,…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The citizens of Cincinnati know one universal truth: that the annual Flying Pig Marathon weekend will bring fun, family, and fulfillment to their city. And with that universal truth comes…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…
Drug development within the rare disease space can be tricky. In many cases, there is a general lack of research and understanding around these conditions; as a result, many…
The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…
The Muscular Dystrophy Association (MDA) held its MDA Clinical & Scientific Conference from March 19-22, 2023 to share research, cutting-edge medical advancements, and clinical care practices within the muscular…
According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…
CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…
Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…
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