The Penn Medicine Orphan Disease Center at the University of Pennsylvania in Philadelphia has announced the recipients of the “2016 Improved Therapies for MPS I” pilot grants. They include two Lysosomal Disease Network physician-researchers: Igor Nestrasil, MD, PhD and Raymond Wang, MD:
• Igor Nestrasil, MD — University of Minnesota
Discovery of brain MRI signatures in infants with severe form of MPS I in the pre-HSCT and post-HSCT stage
• Raymond Wang, MD — CHOC Children’s Hospital (Orange County, California)
Intra-articular Gene Therapy for Canine Mucopolysaccharidosis Type I Joint Disease
• Joseph Anderson, PhD — University of California, Davis
Alpha-L-iduronidase Delivery via Human CD34+ Hematopoietic Stem Cells
• Kim Keeling, PhD — University of Alabama at Birmingham
Identify Drugs to Treat MPS-IH Caused by Nonsense Mutations
• Lachlan Smith, PhD — University of Pennsylvania
Novel therapies to improve bone formation in Mucopolysaccharidosis I Dogs
• Drew Weissman, MD, PhD — University of Pennsylvania
Nucleoside modified mRNA therapy for MPS I
• Carmine Settembre, PhD —
Telethon Institute of Genetics and Medicine (Pozzuoli, Italy)
Modulation of autophagy to treat skeletal features in MPS I
Congratulations to these brilliant researchers!