In a mid-November 2022 press release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. (“CANbridge”), the company shared that its therapeutic option CAN106, which binds to and neutralizes C5 in the…
According to a story from PR Newswire, the pharmaceutical company Saol Therapeutics has recently announced that it has completed its enrollment goals for its phase 3 clinical trial. This trial…
On December 8, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
According to a story from finance.yahoo.com, a new treatment regimen has been approved by the US Food and Drug Administration (FDA) for hepatocellular carcinoma that cannot be treated with surgery.…
An announcement via PR NewsWire by NeuroSense Therapeutics based in Cambridge, Massachusetts carried the results of its multi-dose study of PrimeC to treat amyotrophic lateral sclerosis (ALS) Expectations are…
According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's…
In July 2022, Global Genes, a nonprofit 501(c)(3) organization and global advocate for those with rare diseases, awarded its Health Equity in RARE Patient Impact grants to members of…
According to the National Institute of Neurological Disorders and Stroke, chorea is defined as involuntary movements that are caused by a neurological disorder. It is one of a group of…
According to a press release from the US Food and Drug Administration (FDA), the agency recently approved a new treatment for multiple myeloma, a type of rare blood cancer. The…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
According to a story from mirror.co.uk, Emma Timofte, age six, was diagnosed with a rare disease called incontinentia pigmenti. The diagnosis came when she was only a few weeks old.…
In 2005, The Chronicles of Narnia: The Lion, the Witch, and the Wardrobe brought us into the fantastical world of Narnia. Much like in the novels, the film's stars journeyed to…
On November 15, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. A rare disease is one…
According to a story from MSN, Joe McCauley was diagnosed with Leigh syndrome, a rare disease, last year. Now 30 years old, he was fortunate to survive a bout of…
According to a news release from the U.S. Food and Drug Administration (FDA) in early November 2022, Dupixent (dupilumab) is now approved for the treatment of adults living with…
According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company's investigational therapeutic alvelestat has earned Fast Track designation from the US…
According to a news release on Yahoo! Finance, clinical-stage biotechnology company Freeline Therapeutics Holdings plc ("Freeline") shared that it had initiated dosing in the second cohort of the Phase…
Patrick Freyne recently interviewed three people with myalgic encephalomyelitis (ME) for his column in the Irish Times. Patrick describes the circumstances behind the first known cases of the ME…
On October 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
In late September 2022, Healio reported that the U.S. Food and Drug Administration (FDA) approved bevacizumab-adcd (Vegzelma), a biosimilar of Genentech’s bevacizumab (Avastin). The approval of biosimilars such as…
According to a story from Patient Worthy partner CureDuchenne, the organization has recently launched its CureDuchenne Occupational Therapist Certification Program. This program is intended to give occupational therapists advanced skills…
October 23 is recognized each year as Kabuki Syndrome Awareness Day, a time to help spread awareness about this syndrome among the general public and in the medical field. Kabuki…
According to a story from the Irish Examiner, young people in Ireland are encouraged to watch out for signs of meningitis following an abnormal cluster of fatalities and serious illness…
According to a story from BioPharma Dive, KalVista Pharmaceuticals has halted its clinical trial evaluating an investigational therapy in development for hereditary angioedema. The trial had included a total of…
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