by Peter Ciszewski | May 20, 2024
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research (ORDR), provides an overview of the ORDR and the research they are involved with. The ORDR is focused on multiple programs to improve the efficacy of rare disease research. Their two...
by Peter Ciszewski | Apr 5, 2024
The U.S. Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation for SGT-003, a gene therapy designed for Duchenne Muscular Dystrophy (DMD) treatment. Duchenne Muscular Dystrophy (DMD) is a genetic disorder that leads to...
by Peter Ciszewski | Mar 10, 2024
At the Rare Disease Day symposium held at the National Institutes of Health (NIH), numerous speakers took the stage to share their knowledge about managing and/or advocating for rare disease patients. One of the highlights of the day however, was from a speaker with...
by Peter Ciszewski | Feb 29, 2024
This symposium, led by Ozlem Goker-Alpan, MD of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), reviews the current state of newborn screening (NBS) for lysosomal disorders and the need for newer guidelines to address the many unmet needs....
by Peter Ciszewski | Jan 7, 2024
Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all...
