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…
The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…
According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…
Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…
Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…
According to a story from Patient Worthy partner CureDuchenne, the organization has recently launched its CureDuchenne Occupational Therapist Certification Program. This program is intended to give occupational therapists advanced skills…
Chances are, you’ll never meet anyone like Lizanne, a 22-year-old woman from the Netherlands. Not only is she extraordinarily passionate about storytelling and filmmaking, but she was diagnosed with a…
In the past, myofibers (terminally differentiated post-mitotic cells that help make up muscle tissue) have been implicated in the development and progression of Duchenne muscular dystrophy (DMD). Basically, researchers have…
Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…
September 7 is World Duchenne Day, a day to help spread awareness about Duchenne muscular dystrophy among the general public and in the medical field. In recognition of this day,…
Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
The Orphan Drug designation was created to aid and hasten the development of drugs created for the treatment, diagnosis, or prevention of rare diseases. When the FDA grants this designation,…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
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