Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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…
Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat,…
The treatment of young patients with R/R acute myeloid leukemia (AML) poses a challenge for clinicians. Chemotherapy regimens, such as high doses of cytarabine, fludarabine, and other salvage reduction therapies,…
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…
GRIN2B Disorders are rare changes in the GRIN2B gene, some causing gain of function, some loss of function, and others unusual neurotransmissions, but all resulting in a variety, and a…
Right now, there are minimal therapeutic interventions for people living with idiopathic pulmonary fibrosis (IPF). Because of this, IPF comes with a life expectancy of 3-5 years past diagnosis.…
Clinical trials are helpful tools in deepening our understanding of certain diseases and evaluating and identifying effective treatment options. Unfortunately, these trials don't always garner the necessary information. For…
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…
According to a story from Healio, the rare disease sarcoidosis can affect a number of areas and systems in the body, such as the eyes, skin, lymph nodes, and lungs.…
Last year, the FDA placed a clinical hold on an Investigational New Drug (IND) application for deucrictibant for the on-demand and prophylactic treatment of individuals living with hereditary angioedema…
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…
Takeda presented encouraging results from the Phase 2b trial (NCT05153148) at the November 2023 American College of Rheumatology (ACR) convergence in San Diego. The Phase 2b study evaluated TYK279 which…
Happy holidays to the Vallecalle family, who are rejoicing in the fact their baby girl is cured of the rare disease, hyperinsulinism. Not only that, but they finally get to…
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