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…
When it comes to rare disease, rare isn't actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Prilenia Therapeutics, Waltham, Massachusetts and Naarden, Netherlands is a biotechnology company with a focus on developing therapeutics to treat neurodevelopmental disorders and neurodegenerative diseases. Prilenia presented data this month at…
According to a story from the Baltimore Sun, a childhood home of Henrietta Lacks - a Black woman often dubbed "the mother of modern medicine" - was unknowingly demolished just…
Despite novel treatment advances, hepatocellular carcinoma (HCC) remains difficult to treat. This aggressive cancer is often not found until later stages and not all people with HCC can utilize available…
The primary outcome from the Phase III NETTER-2 clinical trial as reported recently in Globe Newswire, was heralded as extending progression free survival from 8.5 months to 22.8. The participants…
A Baylor College team of researchers headed by author and Associate Professor of Pathology, Liaising Yen, have spent over ten years working on a technology to effectively regulate gene expression.…
In January 2024, the FDA approved Hyqvia (Immune Globulin Infusion 10% [Human]) as a maintenance therapy for adults living with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder. As…
People with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder, now have another treatment option: Gammagard Liquid (immune globulin infusion [human] 10% solution). Healio reports that the Food and…
The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…
For over 20 years, CureDuchenne, a Patient Worthy partner and global nonprofit, has moved steadfastly forward with a mission to find, and fund, a cure for individuals with Duchenne muscular…
Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
According to an article published in Healthcare Dive, a recent study published in the academic journal JAMA indicated that patients receiving treatment in hospitals owned by private equity firms were at an…
On January 18, 2024, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
In December 2023, clinical-stage biopharmaceutical company Savara Inc. announced that the company had launched a serum-based blood test to aid in diagnosing autoimmune pulmonary alveolar proteinosis (aPAP). While aPAP is…
January 24, 2024 is recognized as Moebius Syndrome Awareness Day, a day to spread awareness about the rare disease Moebius syndrome among the medical field and the general public. The…
On December 5, 2023, the Rare Disease Legislative Advocates (RDLA) held an RDLA Caucus briefing. This briefing, held in collaboration with the Rare Disease Congressional Caucus, provided an update on…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
Michael Strahan is well-known for being an American television personality, journalist, and former NFL Defensive End (DE) for the New York Giants. Now, the family man and proud father is…
The drug Aficamten is being developed by Cytokinetics as a pill taken once daily for hypertrophic cardiomyopathy (HCM). It is a genetic disease where due to a thickness in the…
Carisma Therapeutics, the manufacturer of CT-0508, issued a press release stating that researchers are conducting a combination study using CAR-M cell therapy plus Keytruda (pembrolizumab) in the treatment of overexpressing…
Ferroptosis, a form of cell death, destroys microglia cells, which are associated with the brain’s immune system in vascular dementia and Alzheimer’s. Dr. Stephen Back suggested that attention had not…
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