by Peter Ciszewski | Nov 23, 2020
The U.S. Food and Drug Administration (FDA) has approved RNAi drug, lumasiran, to treat primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic condition that is often difficult to diagnose since its main symptom is the development of kidney stones. These...
by Peter Ciszewski | Nov 23, 2020
Pritesh J. Gandhi, PharmD, of Alnylam Pharmaceuticals discusses the company’s hope that lumasiran obtains FDA approval to treat primary hyperoxalaluria type 1 (PH1). A PDUFA date is set for December 3, 2020. PH1 is a rare genetic disease in which excessive oxalate...
by Peter Ciszewski | Nov 23, 2020
Andrew Krivoshik, MD, PhD, Oncology Therapeutic Area Head at Astellas Pharma explains how acute myeloid leukemia (AML) patients have been affected by the Covid-19 pandemic. AML is a rare aggressive cancer of the blood and bone marrow with a low 5-year survival...
by Peter Ciszewski | Nov 22, 2020
Lisa Nachtigall MD, Clinical Director, Neuroendocrine and Pituitary Clinical Center at Massachusetts General Hospital, and Associate Professor at Harvard Medical School provides an overview of Acromegaly. Acromegaly is a rare disorder caused by a noncancerous...
by Peter Ciszewski | Nov 21, 2020
The U.S. Food and Drug Administration (FDA) has approved Zokinvy (lonafarnib) to treat progeria, a rare disease often known as the advanced aging disease. More specifically, the FDA has approved lonafarnib capsules to reduce the risk of death due to Hutchinson-Gilford...
