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…
According to a recent American Society of Clinical Oncology (ASCO) press release, Dr. Jason Westin provided testimony regarding a nationwide shortage of cancer drugs, numbering more than 15 on the…
According to a story from BioPharma Reporter, the pharmaceutical company Candel Therapeutics recently announced that its investigational therapy designated CAN-2409 has earned Fast Track designation from the US Food and…
A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN…
The increasing penetration of digital technologies and the ubiquity of smartphones and the internet have transformed several spheres of our lives. Healthcare is no exception. With technological advances, it has…
In 2013, the Morells welcomed two identical baby girls, Kaylie and Kenzie. The joy they felt soon disappeared as the twins began to slip back from their normal infant and…
Bladder cancer patients and their families have faced years, and even decades, of waiting for a breakthrough in bladder cancer treatment. For some patients, the results of two clinical…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder in which blood cells break apart prematurely. In an effort to bring more effective and targeted treatments…
Neuroblastoma most commonly occurs in children under the age of five. The disease usually affects areas of the adrenal gland (positioned over the kidneys) or areas in the neck, chest,…
Roche has added another failure this year with its drug gantenerumab. As noted in Fierce Biotech, the drug was unable to improve functional or cognitive decline in two Phase…
The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…
Prior to the FDA’s approval this month, nirogacestat (Ogsiveo) received the following FDA designations applicable to the treatment of desmoid tumors: Breakthrough therapy providing a rapid review. Fast track to…
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