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…
There are many conditions which can be cured or at least ameliorated with a stem cell transplant or an organ transplant. But even when the right donor is available, and…
According to a story from Medscape, a new set of guidelines from the Kidney Disease: Improving Global Outcomes (KDIGO) aims to improve and update standards of care for antineutrophilic cytoplasmic…
In the rare disease community, fundraising plays a pivotal role in driving forward research and treatment initiatives, offering vital support to affected families, and guaranteeing access to necessary resources and…
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Out of 103,000 people in the United States waiting for a transplant, almost 88,000 individuals on the waiting list need a kidney transplant. These statistics were recently provided through Organ…
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