Clinical-stage biotechnology company Abeona Therapeutics ("Abeona") is working to develop a novel cell therapy for people living with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder characterized by…
For people with geographic atrophy, also known as advanced dry age-related macular degeneration, slowing vision loss is at the top of the list in terms of goals. Treatments like SYOFOVRE…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
On December 13, 1996, Health Canada issued a policy statement called Priority Review of Drug Submissions which: provided for the "fast-tracking" of eligible New Drug Submissions (NDS) and Supplemental New…
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, advocates, scientists, and more.…
Zeposia (ozanimod), developed by Bristol Myers Squibb, is an orally administered sphingosine 1-phosphate (S1P) receptor that is currently approved for two separate indications: relapsing multiple sclerosis and ulcerative colitis. According…
In April 2024, biotechnology company Vertex Pharmaceuticals ("Vertex") shared via news release that it was advancing its therapeutic candidate inaxaplin (VX-147) into the Phase 3 portion of a Phase 2/3…
Also known as systemic lupus erythematosus (SLE), lupus is a chronic autoimmune disorder in which the immune system mistakenly attacks different parts of the body such as the joints, kidneys,…
According to a story from BioPharma Dive, the US Food and Drug Administration (FDA) has recently approved Beqvez, a gene therapy from pharmaceutical giant Pfizer intended to treat hemophilia B.…
I have lived with chronic pain from dystonia, a neurological movement disorder, for 23 years. The first 5 years my symptoms were so severe that I was barely able to…
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, advocates, scientists, and more.…
Unexpected but positive results from a late-stage clinical trial were announced by Cerevel Therapeutics and reported in BioPharma Dive last week much to the surprise of AbbVie, the company that…
On one side of the equation pharmaceutical companies maintain that if drug prices are restricted it will have a negative impact on innovation. Health economists, on the other hand, maintain…
According to a story from psypost.org, a treatment approach called transcranial direct current stimulation can help alleviate certain aspects of pain associated with Parkinson's disease. Transcranial direct current stimulation (tDCS)…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
In the United States, a rare condition is defined as one affecting fewer than 200,000 people nationwide. But when you consider how many people actually live with a rare condition,…
An American triathlete, Jon Blais, received a diagnosis of amyotrophic lateral sclerosis (ALS) in 2005 when he was 33 years old. The disease is incurable. Jon was told he had…
What’s better than being in warm and sunny Arizona? Getting to learn alongside the amazing primary biliary cholangitis (PBC) community while there! The PBCers organization held its 2024 Patient Education…
Exa-cel Gene-Edited Therapy Vertex Pharmaceutical recently announced that Health Canada has accepted its New Drug Submission and granted Priority Review for Exa-cel, a gene-edited cell therapy, for the treatment of…
Developing an animal model of a disease can play an important role in both helping researchers to better understand that disease, as well as identifying and testing potential therapeutics. Animal…
According to a story published on tctmd.com, patients living with long COVID can experience a wide range of symptoms. One of these symptoms includes chest pain. Scientists weren't certain what…
According to a story from Yahoo! News, Huw Williams, a notable UK radio presenter, was just days away from performing in front of Bruce Springsteen when he was diagnosed with…
In September 2023, the Virginia Department of Health announced that five people across the state died from a rare, serious, and somewhat unusual strain of meningococcal disease. Now, reports Brenda…
On April 30, 2024, the American Kidney Fund (AKF) will be organizing the first annual APOL1-mediated kidney disease (AMKD) Awareness Day. This awareness day will be a time to…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Jennifer Angus, a…
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