According to a press release from Sanofi, platform and poster presentations focused on the company's investigational product candidate avalglucosidase alfa are slated to be made public at the 17th annual…
In a news release from biopharmaceutical company Bristol Myers Squibb, the company shared that the FDA accepted its supplemental New Drug Application (sNDA). Because the sNDA was submitted alongside…
At the start of February, gene therapy company Spark Therapeutics ("Spark") announced that the first participant in the RESOLUTE trial was dosed with SPK-3006. The trial is evaluating the…
A study has recently highlighted the fact that patients who have type 2 diabetes are more susceptible to chronic kidney disease (CKD), and as a result, cardiac issues. This study…
The month of February is recognized annually as Turner Syndrome Awareness Month, which is intended to help spread awareness about Turner syndrome among the general public and in the medical…
Sometimes it can be difficult to study or learn about rare diseases, especially when there are not many patients. For example, only an estimated 30,000 Americans have Castleman disease. Yet…
The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…
Mitochondrial Disease Mitochondrial disease is a rare and progressive condition that currently does not have a cure. The mitochondria is responsible for providing energy to the body. This energy is used…
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