Latest Breaking News On - Orphan disease - Page 7 : comparemela.com
RARE Drug Development Symposium to have daily morning Fireside Chats
globalgenes.org - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from globalgenes.org Daily Mail and Mail on Sunday newspapers.
Leaked reports allege that Penn officials led shameless cover-up to protect Gene Therapy Program
thedp.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from thedp.com Daily Mail and Mail on Sunday newspapers.
Published: May 17, 2021
REDWOOD CITY, Calif., May 17, 2021 (GLOBE NEWSWIRE)
Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company developing novel therapies for diseases caused by chronic glucose imbalance, today announced that management will present at the Oppenheimer Rare and Orphan Disease Summit. The presentation will be available on-demand starting at 8:00 AM ET on Friday, May 21, 2021.
A webcast of the presentation will be available for 90 days on the “IR Calendar” page within the Investors section of the Rezolute website at https://ir.rezolutebio.com/ir-calendar.
About Rezolute, Inc.
Rezolute is advancing novel therapies for diseases caused by chronic glucose imbalance. The Company’s lead clinical asset, RZ358, is in Phase 2b development for treatment of congenital hyperinsulinism (CHI), a rare pediatric endocrine disorder. The Company is also developing RZ402, an orally available plasma kallikrein inhibitor, for the treatment of diabeti
Advicenne receives positive feedback from the US FDA on pathway to approval and amended Phase III study protocol for its treatment of distal renal tubular acidosis (dRTA) FDA accepts amended protocol and Advicenne will resume Phase III trial of its lead product ADV7103 (Sibnayal™) in the US in June 2021FDA guidance provides clear path for market approval of ADV7103 in the United States Paris, France, 17 May 2021 – 6 pm CEST – Advicenne (Euronext: ADVIC), a specialty pharmaceutical company dedicated to developing and commercializing innovative treatments for those suffering from rare renal diseases, announces US Food and Drug Administration (FDA) acceptance of the amended protocol of the Company’s US-based Phase III pivotal trial of ADV7103 (Sibnayal™) for the treatment of primary distal renal tubular acidosis (dRTA). dRTA is an orphan disease of the kidney affecting an estimated 30,000 patients in Europe and 20,000 in the US for which no approved trea
Errant Gene Therapeutics Becomes San Rocco Therapeutics
Errant Gene Therapeutics was founded in 1993, after founder Pat Girondi s son was diagnosed with Thalassemia, a cousin disease to Sickle Cell Anemia.
In 2007, with the help of researchers from Memorial Sloan Kettering, Cornell and National Institute of Health, EGT became the first entity to pass the FDA Recombinant DNA Committee for gene therapy in Sickle Cell Disease and Beta Thalassemia. EGT was the first company to get Orphan Drug Designation for Thalassemia in the US and Europe and first to produce a commercial batch (8-10 patients) of gene therapy for Sickle Cell Disease and Thalassemia. EGT is the company with the longest track record of treating US patients and the only company with experience in both harsh and soft chemotherapeutic prep-regimens.
vimarsana © 2020. All Rights Reserved.