Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the unique challenges faced by patients,…
The Food and Drug Administration has approved the second cancer treatment that will be totally personalized, proving that these kinds of treatments will become a regular thing moving forward. The…
The clock is ticking. If you or someone you love is diagnosed with a rare disease like hypoplasminogenemia, days -- often even hours -- can mean the difference between life…
Patients with hepatocellular carcinoma can look forward to the FDA accelerated approval of nivolumab (Opdivo) after prior sorafenib (Nexavar) treatment. Hepatocellular carcinoma is a common form of liver cancer that cannot be…
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