Latest News - Neuromuscular

Wednesday, August 15, 2018

First-in-Class RNAi Treatment for Hereditary Transthyretin-Mediated Amyloidosis

The US Food and Drug Administration (FDA) has approved patisiran (Onpattro; Alnylam Pharmaceuticals, Cambridge, MA) for treatment of polyneuropathy in adults with hereditary transthyretin-mediated amy…

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Monday, July 30, 2018

Breaching the Blood-Brain Barrier: A Potential New Treatment Modality

In a small, yet groundbreaking, clinical trial, MR-guided focused ultrasound (MRgFUS) (ExablateNeuro; Insightec, Miami FL) was used to open the blood-brain barrier (BBB) safely in 5 persons with mild-…

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Thursday, July 26, 2018

Amyotrophic Lateral Sclerosis the Next Target for Antisense Oligonucleotide Therapy?

Antisense oligonucleotide (ASO) therapy is a new class of treatment of people with illnesses caused by defects in a single gene. In this novel approach, instead of repairing or replacing a gene functi…

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Friday, July 13, 2018

IncobotulinumtoxinA Approved for Treatment of Excessive Drooling

The Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for incobotulinumtoxin A (Xeomin; Merz, Raleigh, NC) for the treatment of chronic sialorrhea, …

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Tuesday, June 26, 2018

BrainStorm Will not Make Investigational Nurown Stem Cell Treatment Available Under Right-to-Try Legislation

BrainStorm Cell Therapeutics Inc. has announced that after careful consideration of the newly enacted right-to-try (RTT) legislation, it will not make stem-cell therapy (Nurown) available to patients …

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Friday, June 01, 2018

BrainStorm to Host Phone Discussion of Right to Try Legislation and Stem Cell Therapy June 7th

Brainstorm Cell Therapeutics Inc (New York, NY) will host a phone call on June 7th at 8:15 AM EDT to provide interested parties an opportunity to engage with the company regarding new “right-to-…

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Thursday, April 26, 2018

OnabotulinumtoxinA: Safety and Efficacy for Treating Children With Spasticity in Cerebral Palsy

At the American Academy of Neurology (AAN), researchers Heakyung Kim, Jill Meilahn, Chengcheng Liu, Henry G. Chambers, and Rozalina Dimitrova presented results evaluating the efficacy and safety of on…

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Wednesday, April 04, 2018

First Enrollment in Open Label Extension of Phase 2 Clinical Trial of AMX0035 for Treatment of Amyotrophic Lateral Sclerosis

The first patients to complete the phase 2 clinical trial of AMX0035 (Amylyx; Cambridge, MA), a combination of phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), for amyotrophic lateral sclero…

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Wednesday, March 21, 2018

Mutations in the Gene for Muscle Protein Kinesin A Cause of Amyotrophic Lateral Sclerosis

As published in today’s issue of the journal Neuron, researchers have identified specific mutations in the kinesin family gene, KIF5A, that cause amyotrophic lateral sclerosis (ALS) (Neuron. 201…

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Friday, March 16, 2018

Subcutaneous Immune Globulin Approved for Treatment of Patients With Chronic Inflammatory Demyelinating Polyneuropathy

Already approved in 51 countries for some immune deficiencies, subcutaneous immune globulin (IG) (Hizentra; King of Prussia, PA) has now been approved by the Food and Drug Administration (FDA) for tre…

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Thursday, March 15, 2018

Positive Results for Phase 2 Proof-of-Concept Study for Congenital and Childhood Onset Myotonic Dystrophy Type 1

The results of a phase 2 proof-of concept study of AMO-02, an investigational therapy for childhood-onset myotonic dystrophy type 1 were presented at the Muscular Dystrophy Association Meeting this we…

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Tuesday, March 13, 2018

Phase I/IIa Clinical Trial of Stem Cell Therapy for Amyotrophic Lateral Sclerosis Begins in Israel

Approval has been granted by the Israel Ministry of Health’s Supreme Committee for Kadimastem (NessZiona, Israel) to immediately begin a phase I/IIa clinical trial of their stem cell therapy to …

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Thursday, March 01, 2018

Biosimilar Being Developed for Botox

In a transatlantic collaboration, Mylan N.V. (Pittsburgh, Pennsylvania) and Revance Therapeutics, Inc. (Hertsfordshire, England) have reached an agreement to collaborate on the development and commerc…

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Wednesday, February 21, 2018

Microglia May Promote Recovery of Motor Neurons in Amyotrophic Lateral Sclerosis

In findings published this week in Nature Neuroscience, researchers Krista J. Spiller, Clark R. Restrepo, Tahiyana Khan, and others described creation of a mouse model of amyotrophic lateral sclerosis…

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Wednesday, February 07, 2018

Results from Phase 2 Trial of Stem-Cell Treatment for Amyotrophic Lateral Sclerosis to Be Presented at American Academy of Neurology 2018

Brainstorm Cell Therapeutics Inc. announced that they will present results from the NurOwn phase 2 randomized, double-blind, placebo-controlled study, showing ALSFRS-R subgroup analyses and micro-RNA …

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