by Peter Ciszewski | May 14, 2022
The U.S. Food and Drug Administration (FDA) has approved an oral form of edaravone (Radicava ORS), for the treatment of amyotrophic lateral sclerosis (ALS), a rapid neurodegenerative disease. An intravenous (IV) formulation of edaravone was approved in 2017. ALS, also...
by Peter Ciszewski | May 12, 2022
Sherif El-Harazi, MD, Ophthalmologist and Medical Director and Founder of Lugene Eye Institute and Global Research Management, discusses cyclosporine ophthalmic emulsion 0.1% eye drops (Verkazia) which is now available in the United States for the treatment of...
by Peter Ciszewski | May 10, 2022
Giacomo Chiesi, MBA, Head of Chiesi Global Rare Diseases, discusses a white paper titled, “The Burden of Rare Diseases: An Economic Evaluation,” based on the results of a study which demonstrate that rare diseases impose substantial economic burden...
by Peter Ciszewski | May 9, 2022
Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine, highlights the latest research about Mucopolysaccharidosis type II (MPS II; Hunter syndrome) presented at WORLDSymposium 2022. This free CME program provides...
by Peter Ciszewski | May 6, 2022
Axel Bolte, MSc, MBA, Co-Founder, President, and Chief Executive Officer, Inozyme Pharmaceuticals, discusses the positive preliminary data from the first 3 patients treated in the phase 1 portion of the phase 1/2 clinical trial of INZ-701 in ENPP1 deficiency...
