by Peter Ciszewski | Jul 24, 2018
Emmaus Life Sciences announced that the New England Journal of Medicine (NEJM) has published the results of its 48-week phase 3 clinical trial of Endari (L-glutamine oral powder) which supported the FDA approval in 2017 to reduce the acute complications of sickle cell...
by Peter Ciszewski | Jul 23, 2018
There seems to be some good news on the horizon for individuals with Charcot-Marie-Tooth (CMT) disease. Acceleron Pharma announced positive preliminary results from Part 1 of the Phase 2 clinical trial with ACE-083 in patients with CMT disease at the Peripheral Nerve...
by Peter Ciszewski | Jul 22, 2018
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Yuval Cohen, PhD, CEO of Corbus Pharmaceuticals discusses the appointment of John K. Jenkins, MD to its Board of Directors and the upcoming data from open-label...
by Peter Ciszewski | Jul 22, 2018
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company, provides an overview of his platform. Baebies was founded in 2014, following the...
by Peter Ciszewski | Jul 22, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses how physicians need to consider rare diseases for some of their more challenging diagnositic cases. Rare diseases are problematic on many fronts: (1)...
