by Peter Ciszewski | Mar 28, 2023
The U.S. Food and Drug Administration (FDA) has approved leniolisib (Joenja) to treat persons 12 years age an older with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS). APDS is a rare primary immunodeficiency caused by genetic variants in either...
by Peter Ciszewski | Mar 22, 2023
Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) provides an overview of recent advances in gene therapy. As Dr. Marks states, we are in a very interesting time for gene therapy...
by Peter Ciszewski | Mar 16, 2023
Scott Gray, Chief Executive Officer of Clincierge, discusses barriers to clinical trial participation and how his company reduces them. As Mr. Gray explains, common barriers facing clinical trial participation include language barriers, travel and lodging logistics,...
by Peter Ciszewski | Mar 8, 2023
Raymond Wang, MD, Metabolic Specialist at Children’s Hospital of Orange County, gives an update on children with for mucopolysaccharidosis type I (MPS I) given gene therapy. MPS I is a lysosomal disorder caused by a deficiency in the enzyme,...
by Peter Ciszewski | Mar 6, 2023
Jana von Hehn, PhD, Chief Scientific Officer of the Rett Syndrome Research Trust, discusses current strategies for managing Rett syndrome. Rett syndrome is a multisystem disorder that primarily affects girls. Only in rare cases are boys affected (who may...
