FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of amyotrophic lateral sclerosis (ALS). The expanded approval for use with PEG feeding tubes will enable patients who not only have difficulty swallowing but also require . . .

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Amylyx Pharmaceuticals Announces Positive Results from Phase 2 Study of AMX0035 for Treatment of ALS

On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment with the company’s investigational therapy, AMX0035, in patients with amyotrophic lateral sclerosis (ALS). The study met its primary endpoint, showing a statistically significant slowing of . . .

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New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease

MDA’s Medical Education department was established in response to the growing need for clinician education in neuromuscular disease (NMD). We are pleased to present four webinars that cover newborn screening (NBS) and early intervention, genetic testing, and best practices in multidisciplinary care. Our Medical Education department, together with key opinion leaders in the NMD field, . . .

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Kadimastem Announces Positive Interim Results from Phase 1/2a Study of AstroRx in ALS

Israel-based Kadimastem Ltd. announced positive interim results from the first cohort of its phase 1/2a clinical trial evaluating the safety, tolerability, and preliminary efficacy of AstroRx in patients with amyotrophic lateral sclerosis (ALS). Results presented at the 7th International Stem Cell Meeting of the Israel Stem Cell Society held recently in Tel Aviv, Israel, showed that patients . . .

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New MDA Educational Program Offers a 360-Degree View of ALS Care

MDA’s new Medical Education department was established in response to the growing need for clinician education in neuromuscular diseases. We are pleased to present our first program highlighting amyotrophic lateral sclerosis (ALS): a PowerPoint slide deck. ALS is a motor neuron disease that leads to the widespread loss of muscle use. Patients diagnosed with ALS . . .

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At a Cedars-Sinai Care Center, Dr. Robert Baloh Looks Under — and Ahead of — the Microscope for CMT and ALS

Even though the Martinez family lives in Burbank, Calif., within an hour’s drive to Los Angeles’ Cedars-Sinai Medical Center, their road to the hospital’s specialized clinics — to Dr. Robert Baloh and his work there — was a long one. Daniel and Gladis Martinez have two daughters: Genesis, 15, and Isabella, 14, born just 10 . . .

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Participants Needed for a Pilot Study of a New Diagnostic Device

Dr. Perry Mansfield at the SENTA Clinic in San Diego, Calif., is looking for healthy individuals and patients with neuromuscular disease who have documented neurologic pharyngeal muscle dysfunction (dysfunction of the voluntary muscles that form the pharynx) to participate in a pilot study. This study will help to prove the diagnostic consistency of transmembrane electromyography . . .

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The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS

The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of . . .

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2109 Clinical & Scientific Conference Highlight: Healthcare Technology Moves Out of the Clinic, Into the Pocket

Computers, laptops, tablets, and smartphones have become ubiquitous features of our daily lives — but not of our healthcare. At least, not yet. But a group of physicians at MDA’s 2019 Clinical & Scientific Conference made a few convincing cases for increased use of personal electronics in healthcare and clinical trials. Dr. James Berry of . . .

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