Views & Analysis Duchenne's disease: A New Hope Rare diseases, exceptional people: Filippo Buccella has battled for 25 years to improve care for children with
Views & Analysis LHON in the family: living under the spectre of an hereditar... It’s hard to imagine suddenly losing your sight in your 20s, but that’s what happened to Richard Wheeler when he developed
Partner Content Partner Content Raising awareness of LHON for Rare Disease Day 2018 As Rare Disease Day approaches on 28 February, Santhera has created an infographic explaining the signs and symptoms of the rare disease Leber’s hereditary optic neuropathy (LHON), a progressive eye disease that results in blindness.
News Catalyst to re-submit rare disease drug after trial success Already approved in EU, Catalyst aiming for FDA filing next year.
Views & Analysis Out of the darkness: new gene therapies for eye diseases US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.
Views & Analysis Shedding light on LHON: the rare blindness affecting young a... LHON is a frequently misdiagnosed condition that needs faster diagnosis.
News Ipsen' Bylvay fluffs its lines in biliary atresia trial Ipsen's IBAT inhibitor Bylvay has failed a trial in biliary atresia, a rare and serious liver disease that affects babies and has limited treatments.
Digital Sponsored Discover a new era of healthcare panel engagement: Grounded ... A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.
