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…
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…
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…
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…
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.…
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…
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,…
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…
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…
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…
Approximately 540,000 individuals in the United States have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD). The disease causes persistent and rapidly accumulating cysts to grow in the patient’s…
Last fall, a new vaccine for respiratory syncytial virus (RSV) intended for pregnant people was made available to the public. Physician Sarah Turner, who was used to getting some pushback…
Previously, Patient Worthy reported on research focused on the use of a pig kidney in a transplant operation involving a human patient. Richard (Rick) Slayman was released from the hospital…
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
According to a press release from the US Food and Drug Administration (FDA), the agency has approved the medication resmetirom (marketed as Rezdiffra) as the first-ever treatment intended to treat…
Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…
During the past three years two drugs (Aduhelm and Leqembi) that were designed to slow the progression of Alzheimer’s disease, have been approved. A verdict by the FDA on a…
Recently, the US Food and Drug Administration (FDA) approved a label expansion for the monoclonal antibody dupilumab (marketed as Dupixent) in the treatment of a rare disease called eosinophilic esophagitis…
The month of April is recognized as Sjögren's Awareness Month, a time for spreading awareness about the autoimmune disease Sjögren's syndrome in the medical community and among the general public.…
While there is no cure for myasthenia gravis, this disorder can be managed with treatment. Current treatment options include a thymectomy, C5 protein inhibitors, and intravenous immune globulins (among others).…
Pulmonary arterial hypertension (PAH) is a rare, chronic, and progressive disorder characterized by high blood pressure in the lungs. This causes pulmonary arteries to thicken or harden, which eventually puts…
Jenny Decker began her solo voyage in her 1983 Bristol 35.5 Tiama from Honokōhau, Hawaii on June 28, 2023 (see Patient Worthy articles Part 1 and Part 2.) Jenny hopes…
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