by Brad Dunn | Jan 29, 2021
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, offers advice to parents of children newly diagnosed with Niemann-Pick Type C (NPC). NPC is a disabling neurogenetic...
by Brad Dunn | Jan 29, 2021
Sanjay Shukla, MD, the president and CEO of aTyr Pharma, describes how his company works in close conjunction with patient groups — including the Foundation for Sarcoidosis Research — to develop its ATYR1923 therapy for pulmonary sarcoidosis. Pulmonary...
by Peter Ciszewski | Jan 28, 2021
Prilenia Therapeutics recently announced the enrollment of the first participant in Europe for its phase 3 clinical trial in Huntington disease patients. The PROOF-HD trial is a pivotal trial being conducted in North America and Europe in collaboration with the...
by Peter Ciszewski | Jan 28, 2021
Nancy Kuntz MD, from the Northwestern University Feinberg School of Medical, discusses the importance of genetic counseling for a family with a child with spinal muscular atrophy (SMA). SMA is a rare neuromuscular disorder caused by mutations in the SMN1 gene...
by Peter Ciszewski | Jan 28, 2021
Auris Huen, MD, PharmD, MD Anderson Cancer Center, Department of Dermatology discusses a multidisciplinary approach to treating cutaneous T-cell lymphoma (CTCL). Although treatment of early-stage CTCL is often managed primarily by dermatologists, advanced disease is...
