Muscular Dystrophy Association Awards Eight Grants Totaling More Than $2 Million for ALS Research

Today, the Muscular Dystrophy Association (MDA) announced the awarding of eight new grants totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which muscles become weak and eventually nonfunctional. Since its inception, MDA has invested more than $165 million in ALS research, and in the last five years . . .

Read More

FDA Awards Fast Track Status to RNS60, Revalesio’s Investigational Therapy for ALS

On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60.   Fast Track status helps to shorten the time it takes to develop and approve a drug that’s been designed to treat a serious condition and fill an unmet . . .

Read More

Clinical Trial Alert: Brainstorm Cell Therapeutics Seeks Participants for a Phase 3 ALS Study

Researchers at Brainstorm Cell Therapeutics are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a Phase 3 study designed to help researchers evaluate the effects of mesenchymal stromal stem cells secreting neurotrophic factors (MSC-NTF cells) on disease progression in patients with ALS.  In ALS, motor neurons degenerate or die, and stop sending . . .

Read More

MDA Awards Venture Philanthropy Grant to AcuraStem to Develop Treatment for ALS

The Muscular Dystrophy Association has awarded an MDA Venture Philanthropy (MVP) grant totaling $300,000 over two years to AcuraStem to support preclinical development of a novel small molecule therapeutic for amyotrophic lateral sclerosis (ALS). The treatment has the potential to be transformative for a broad range of ALS patients. MVP grants are awarded to researchers . . .

Read More

Clinical Trial Alert: Orphazyme Seeks Participants for a Phase 3 ALS Study

Researchers at Orphazyme are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the ORARIALS-01 Phase 3 study. The study is designed to help researchers evaluate the effects of arimoclomol on disease progression in patients with ALS.  In ALS, motor neurons degenerate or die and stop sending messages to the muscles, which gradually . . .

Read More

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

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 . . .

Read More

Biohaven Pharmaceuticals Announces FDA Has Accepted Its 505(b)(2) New Drug Application to Review BHV-0223 for Treatment of ALS

On Nov. 26, Biohaven Pharmaceuticals announced that the Food and Drug Administration (FDA) has accepted its 505(b)(2) New Drug Application (NDA) to review BHV-0223, a sublingual (placed under the tongue) formulation of riluzole for the treatment of patients living with amyotrophic lateral sclerosis (ALS). In submitting its NDA using the 505(b)(2) pathway, Biohaven was able . . .

Read More

FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS

On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .

Read More

MDA and Answer ALS Partner to Develop the Most Comprehensive ALS Dataset to Date

MDA recently announced that it has awarded a research infrastructure grant totaling $550,000 to Jeffrey Rothstein, M.D., Ph.D., Professor of Neurology and Neuroscience and Director, Robert Packard Center for Answer ALS Research, Johns Hopkins University School of Medicine. Answer ALS is a nationwide consortium assembling one of the most comprehensive clinical, genetic, molecular, and biochemical . . .

Read More

Clinical Trial Alert: Orion Pharma Seeks Participants for a Phase 3 ALS Study

Researchers at Orion Pharma are looking for participants with ALS (amyotrophic lateral sclerosis) to participate in the REFALS Phase 3 study. The study is designed to help researchers evaluate the effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS. Participants will be randomized to receive either levosimendan or placebo to determine whether . . .

Read More