Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these conditions, and current research advances may soon shift the therapeutic landscape for eye health. However, many obstacles remain in place, as this Special Feature discusses.
Study Overview
Official Title
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Brief Summary
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS).
Objectives
- Summarize two types of Wolfram syndrome and related disorders.
- Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
- Emphasize the need for cooperation with patient organizations and industry partners to support the development of new therapies.
Venue: Sir Christopher Wren Hotel, Thames Street, Windsor, Berkshire, SL4 1PX
Invite only!
For more information please get in touch with Stephanie@thesnowfoundation.org
Dr. Giulio Frontino and Dr. Maurizio Delvecchio of IRCCS San Raffaele Hospital, Milan, Italy. Unified Care for Diverse Challenges: Crafting National Guidelines for WS from Diagnosis to Care. You can listen to the presentation by following the link provided. https://youtu.be/PssLjWZgsFE
Head to the link to listen to it now. https://youtu.be/zQ-WrsgEi3o
Dr. Fumihiko Urano’s research updates along with Amylyx Pharmaceuticals’ update on their ongoing clinical trial involving AMX0035. It’s an incredible chance to learn more about the latest advancements in this field. Listen to the updates by clicking on the link provided: https://youtu.be/WCUNVi90EGo
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