Clinical Trial Alert: Researchers at Neurogene Seek Participants for a CMT4J Natural History Study

Researchers at Neurogene are seeking participants living with Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials. This five-year study will consist of 10 visits every six months. Each visit . . .

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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 . . .

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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 . . .

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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 . . .

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Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

On Jan. 3, Wave Life Sciences announced that its upcoming Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, has been selected by the U.S. Food and Drug Administration (FDA) for its complex innovative trial designs (CID) pilot program.  Wave . . .

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FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .

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Ra Pharmaceuticals Announces Positive Results from Phase 2 Trial of Zilucoplan in Patients with Generalized Myasthenia Gravis

On Dec. 10, Ra Pharmaceuticals Inc., based in Cambridge, Mass., announced positive results from a Phase 2 clinical trial designed to evaluate zilucoplan (RA101495) for treating generalized myasthenia gravis (gMG). Patients who received the drug had significant reductions in both measured endpoints, the Quantitative Myasthenia Gravis (QMG) score and the Myasthenia Gravis — Activities of . . .

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