Tag: Ionis

News, Research

Clinical Trial Alert: Phase 3 Study of ION363 in Individuals with ALS with Fused in Sarcoma Mutations (FUS-ALS)

MDA Staff

Researchers at IONIS Pharmaceuticals are seeking individuals living with amyotrophic lateral sclerosis (ALS) with fused in sarcoma mutations (FUS-ALS) to participate in a phase 3 clinical trial to determine the efficacy of the investigational drug ION363. Drug efficacy will be determined by measuring clinical functioning and survival in trial participants.   The study consists of two parts, . . .

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News, Research

Do You Know ALS? Read About the Latest Research.

Jeanene Swanson

It’s a hard fact that only three medications have been approved to treat amyotrophic lateral sclerosis (ALS) in nearly 25 years. Fortunately, research in ALS has exploded in the past decade, and new technologies have enabled the development of gene-targeting therapies such as gene replacement, gene silencing, and gene editing. MDA has always been committed . . .

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News, Research

Biogen Announces Positive Phase 1 Results, Plan to License and Develop Ionis Pharmaceuticals’ Investigational Therapy BIIB067 for Familial ALS

Jeanene Swanson

On Dec. 6, Biogen Inc. announced positive Phase 1 clinical trial results and its decision to license and develop partner Ionis Pharmaceuticals’ BIIB067 (IONIS-SOD1RX). BIIB067 is an investigational therapy for treating amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) mutations, a subtype of familial ALS that makes up 2 percent of all ALS . . .

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