by Peter Ciszewski | Feb 18, 2021
Dustin Armstrong, PhD, president of Parasail, LLC, talks about the drug, VAL-1221, for the treatment of Pompe disease. Pompe disease is a lysosomal disorder that leads to reduced levels of acid alpha-glucosidase (GAA), which in turn leads to a buildup of...
by Peter Ciszewski | Feb 18, 2021
Michael Levy, MD, PhD, Associate Neurologist at Massachusetts General Hospital, provides an overview of Neuromyelitis Optica Spectrum Disorder (NMOSD). NMOSD is an autoimmune disease often characterized by sporadic and repeated inflammatory flare ups that can...
by Peter Ciszewski | Feb 17, 2021
Stephanie Cherqui, PhD, from the University of California, San Diego, talks about the latest data testing hematopoietic stem cell gene therapy as a treatment for cystinosis. Cystinosis is a rare lysosomal storage disorder characterized by the accumulation of...
by Peter Ciszewski | Feb 16, 2021
Morie Gertz, MD, Professor of Medicine at the Mayo Clinic is a specialist in diagnosing and treating amyloid transthyretin (ATTR) amyloidosis. One of the biggest challenges with this rare condition is getting a quick diagnosis. Many clinicians who see these...
by Peter Ciszewski | Feb 12, 2021
Pasi A. Jänne, MD, PhD, from the Dana-Farber Cancer Institute, discusses the efficacy and safety of mobocertinib in previously treated patients with EGFR exon 20-mutated non–small cell lung cancer (NSCLC). Patients with EGFR-mutant NSCLC make up only about 1-2%...
