Gene therapy offers hope for patients with rare vision loss

A groundbreaking gene therapy treatment has shown remarkable success in improving vision for patients with Leber congenital amaurosis (LCA1), a rare inherited condition that causes severe vision loss. The therapy, which targets a specific genetic mutation, has resulted in significant improvements in vision for some patients.

https://www.vanguardngr.com/2024/09/gene-therapy-offers-hope-for-patients-with-rare-vision-loss/

Ogundipe, Sola. (2024, September 29). Gene therapy offers hope for patients with rare vision loss. Vanguard.

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Diabetes Quality Measures Have Challenges But Also Successes

Quality measures are a tool to drive healthcare toward value-based care, and in diabetes care these measures can improve diabetes management and prevent downstream conditions, such as cardiovascular disease. However, there are multiple hurdles that healthcare organizations face when implementing quality measures for diabetes care, explained Sean M. Oser, M.D., M.P.H., associate professor in the Department of Family Medicine at the University of Colorado School of Medicine, in a Managed Healthcare Executive K-Cast video series.

https://www.managedhealthcareexecutive.com/view/diabetes-quality-measures-have-challenges-but-also-successes

MHE Staff (2024, October 3). Diabetes Quality Measures Have Challenges But Also Successes. Managed Healthcare Executive.

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Is gene therapy the next big step in vision loss treatment?

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.

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AMYLYX Phase 1/2 Clinical Trial

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.

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Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 2 Study of AMX0035 for the Treatment of 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).

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Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 Helios Clinical Trial

Objectives

  1. Summarize two types of Wolfram syndrome and related disorders.
  2. Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
  3. Emphasize the need for cooperation with patient organizations and industry partners to support the development of new therapies.

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GLP-1 drugs protect brain health by improving neurovascular function and reducing inflammation

GLP-1 drugs protect brain health by improving neurovascular function and reducing inflammation

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