A new film is set to premiere tomorrow, and it depicts the life of Jeff Golub. This famous musician was known for his talent on the guitar, but what many…
Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…
Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…
According to Healio, the FDA recently granted Orphan Drug designation to nadunolimab (CAN04), a treatment candidate for patients with pancreatic cancer. Orphan Drug designation is given to drugs or biologics…
Patient Worthy recently had the privilege to view a selection of short films about rare disease at Biotech Week Boston. These films highlight the stories of families and patients as…
A recent study conducted by the Penn State College of Medicine revealed that taking glucagon-like peptide-1 receptor (GLP-1R) agonists six months before COVID-19 infection decreases the risk of a severe…
A new collaboration between the University of Texas's MD Anderson Cancer Center and the Rare Cancer Research Foundation aims to solve a problem that has been a major obstacle for…
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