FDA Clears MCO-010 Gene Therapy as IND for Stargardt Macular Degeneration
January 26th 2022A gene therapy for retinal degenerative diseases receives an investigational new drug (IND) clearance from the FDA to move forward with the Phase 2 trial of the multi-characteristic opsin (MCO-010) therapy.
Read More
New Data Examines Mortality Risk and Allopurinol Use in Patients with Gout and CKD
January 24th 2022Contrary to recent research that indicated a higher risk of death associated with escalating doses of allopurinol for chronic kidney disease, investigators announce no association between allopurinol initiation and increased mortality among patients with gout and CKD.
Read More
New Data Reveals a Stem-Cell Gene Therapy for Metachromatic Leukodystrophy
January 24th 2022Since an effective treatment for the rare disease metachromatic leukodystrophy had not previously been distinguished, investigators analyzed data on the safety and efficacy of arsa-cel, a gene therapy.
Read More
Tim Duong, PhD, Wouter Hoogenboom, PhD: What is Known About COVID-19 and SCD
January 17th 2022After conducting and extensive literary review on all eligible data regarding COVID-19 in patients with sickle cell disease or trait, experts discuss what is known and what needs to be better understood about the pandemic's effect on this population.
Read More