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…
Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…
Ronald Acuña Jr. is a professional baseball player with the Atlanta Braves. The powerhouse player is known for being an NL MVP and for becoming the first player in Major…
There are over 30 subtypes of congenital muscular dystrophy (CMD), or muscular dystrophies that are present at birth. These rare genetic disorders often manifest in hypotonia (low muscle tone) and…
The Sudden Arrhythmia Death Syndromes (SADS) Foundation - a Patient Worthy partner - was founded in 1991 to provide support and care for people affected by heart arrhytmia conditions that…
The overwhelming majority of rare diseases and conditions have a genetic basis. This means that the disease is the result of a genetic abnormality such as a mutation. These abnormalities…
In early March 2024, Tyler Patchen reported that the U.S. Food and Drug Administration (FDA) approved both intravenous and subcutaneous formulations of Tyenne (tocilizumab-aazg) for use in a variety of…
According to a story from Healio, encouraging results from a phase 2 trial could herald a new standard of treatment for metabolic dysfunction-associated steatohepatitis (MASH). The investigational therapy is called…
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