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January 19, 2021January 19, 2021

Radicava Out-of-pocket Assistance Program Made Easier

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Mitsubishi Tanabe Pharma America announced changes to enhance its out-of-pocket assistance program for people with amyotrophic lateral sclerosis (ALS) using Radicava (edaravone). The changes, aimed at making the program ... Read more

January 18, 2021January 18, 2021

Honoring Stephen Hawking, Answer ALS Opens Data Portal to Scientists

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To mark what would have been the 79th birthday of the theoretical physicist Stephen Hawking, who lived with amyotrophic lateral sclerosis (ALS) for 55 years, Answer ALS is providing ... Read more

January 15, 2021January 15, 2021

Mexiletine May Lessen Motor Neuron ‘Excitability’ to Ease Cramps, Small Trial Suggests

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Mexiletine, a heart medication used off-label to treat muscle cramps due to amyotrophic lateral sclerosis (ALS), was found to be well-tolerated and to stabilize the “hyperexcitability” of motor neurons ... Read more

January 14, 2021January 14, 2021

Pooling Imaging Data May Make Multi-center Clinical Trials Easier

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Imaging data of brain inflammation in people with amyotrophic lateral sclerosis (ALS) that is obtained using different tracers and imaging equipment can be combined using a technique called the ... Read more

January 13, 2021January 13, 2021

PrimeC Earns Orphan Drug Designation in Europe

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The European Medicines Agency has granted orphan drug designation to PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS). PrimeC, developed by NeuroSense Therapeutics, also has received orphan drug ... Read more

January 12, 2021January 12, 2021

First Patient Enrolled in Pridopidine Arm of the HEALEY ALS Platform Trial

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A pivotal clinical trial evaluating pridopidine for the treatment of amyotrophic lateral sclerosis (ALS) has enrolled its first participant, Prilenia Therapeutics, the medicine’s maker, announced. The Phase 2/3 study, ... Read more

January 11, 2021January 11, 2021

COVID-19 Safety Measures Announced for Biomarker Study

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Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s ... Read more

January 8, 2021January 8, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

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The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 8, 2021January 8, 2021

CNM-Au8 May Be Source of Motor Neuron Gains Being Seen in Phase 2 Trial

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Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health ... Read more

January 7, 2021January 7, 2021

Spinogenix Wins Grant to Advance Testing of Potential Oral Therapy

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Spinogenix has been awarded a research grant from the U.S. Department of Defense (DOD) to further test its lead compound as a potential treatment of amyotrophic lateral sclerosis (ALS), ... Read more

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