Type 1 narcolepsy, formerly known as narcolepsy with cataplexy, is a chronic neurological disorder characterized by excessive daytime sleepiness and cataplexy, or the sudden loss of muscle tone that is…
Made with fresh watermelon juice, vodka, triple sec, and lemon-lime soda, a Watermelon Crush is exactly as bubbly, bright, and refreshing as you want from your ideal summer sipper. And…
On February 28th, 2024, the first-ever White House Rare Disease Forum took place. Hosted by the White House Office of Science and Technology Policy, this forum provided a platform for…
Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…
May 2 is Curly Hair Day—a day to celebrate the beauty of waves, coils, and curls. But for one fifth-grader in Canada, Curly Hair Day stands as a way for…
Angelman syndrome begins at birth and although symptoms vary, most children exhibit common developmental delays such as: • Infants showing an inability to support their head • Unable to pull…
In the United States, Orphan Drug designation comes with benefits and incentives such as fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is…
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.…
It has been over four years since our lives were irreparably changed by the onset of the COVID-19 pandemic. With hundreds of millions of cases globally, and millions of associated…
In 2010, K. Pattie Figueroa of the Spencer Fox Eccles School of Medicine at University of Utah was approached by a family whose child had spinocerebellar ataxia type 4 (SCA4).…
The cause of multiple sclerosis, an autoimmune disorder impacting the nervous system, isn't well understood. Genetics, infectious agents, and environmental factors seem to all play a role, but researchers aren't…
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.…
Acknowledgment: This patient story is sponsored by Chiesi USA and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
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…
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