In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
In a recent press release, biopharmaceutical company Akari Therapeutics ("Akari") announced the publication of data on intravitreal nomacopan for patients with autoimmune uveitis. The data was sourced from a 2-year…
Recently, the FDA expanded treatment indications for Gavreto (pralsetinib). According to MedPage Today, the drug was previously indicated for the treatment of patients with lung cancers with RET gene fusions. Now, this…
In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
A review of COVID-19 data from the UK shows that patients with rare autoimmune rheumatic disease (RAIRD) face a higher mortality risk, according to an article in Healio. The study,…
Xavier Alford told BBC News how he vividly recalls the day, twelve years ago, when he was told by his doctor that he had a very rare disease. His doctor…
A study in the European Medical Journal aims to confirm the safety profile of vedolizumab, a treatment for inflammatory bowel disease (IBD). Specifically, they focused on acute infusion reactions. It…
A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly…
According to a story from Hematology Advisor, a recent study released in Scientific Reports indicates that gene expression has the potential to predict both primary resistance and treatment response in patients living…
Overall, Galapagos works to develop small-molecule treatments for patients with a variety of conditions. In a recent press release, the company reported positive top-line data from the Phase 2 PINTA…
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