by Peter Ciszewski | May 11, 2020
Shannon Palmatier has two sons with progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which children are unable to properly remove bile acids from their liver. The disease is a genetic disorder that follows an autosomal...
by Peter Ciszewski | May 9, 2020
Cyndi Frank, of the Gaucher Community Alliance explains her long diagnostic journey.
by Peter Ciszewski | May 9, 2020
The U.S. Food and Drug Administration (FDA) has approved the kinase inhibitor, Retevmo (selpercatinib), to treat people with three cancers that have a mutation or fusion in the RET gene. The three cancers are: Non-small cell lung cancer (NSCLC) that has spread...
by Peter Ciszewski | May 8, 2020
Ovid Pharmaceutics announced their novel delta (δ)-selective GABAA receptor agonist, gaboxadol (OV101), was shown to have a significant improvement in behaviour of young adults with Fragile X syndrome. Fragile X syndrome is an inherited form of intellectual disability...
by Peter Ciszewski | May 7, 2020
Remdesivir in adults with severe COVID-19: a randomised, double-blind, placebo-controlled, multicentre trial Yeming Wang et al published their clinical trial set in Hubei, China showed the use of remdesivir to not have a statistically significant benefit. This is in...
