Happy Father's Day weekend! While Father's Day can be complicated in a rare family, rare parents deserve a round of applause. This week we have a Father's Day tribute from…
The U.S. Food and Drug Administration has granted the experimental drug A4250 rare paediatric disease designation for the treatment of progressive familial intrahepatic cholestasis. The full article can be found…
According to a story from pm360online.com, the speciality pharmaceutical company Chiesi USA and the biotech company Holostem Terapie Avanzate S.r.l. of Italy recently announced that the U.S. FDA recently granted orphan…
I've known some wonderful fathers throughout my life. My own father was a gem. My siblings agree. However, it was not until I became immersed in the rare disease world…
Newron Pharmaceuticals S.p.A (Newron) is a biopharmaceutical company specializing in developing inventive therapeutic options. They focus primarily on conditions related to the nervous system. According to bioportfolio.com, Newron announced on…
According to BioPortfolio, there was recently an announcement about preclinical data for SYNB1618, which is a Synthetic Biotic treatment that is being assessed right now in a Phase 1/2a clinical trial in…
Many people are familiar with Carl Zimmer as a science columnist from The New York Times. His columns and research dug deeply into the field of genetic research and gene…
According to EurekAlert!, a study at Virginia Commonwealth University has potentially pinpointed the so called "Achilles heal" of a certain type of deadly brain cancer: glioblastoma multiforme. Glioblastoma, or glioblastoma…
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
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