Is There a Right or Wrong in the Debate Between Patients and Manufacturers to Release Drugs Through Compassionate Use?
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Is There a Right or Wrong in the Debate Between Patients and Manufacturers to Release Drugs Through Compassionate Use?

  Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen,…

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New Hope for ALS and Friedreich’s Ataxia Patients Through Viral Vector Transports
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New Hope for ALS and Friedreich’s Ataxia Patients Through Viral Vector Transports

A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…

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Masitinib Studies Suspended Over Ischemic Heart Disease Risk
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Masitinib Studies Suspended Over Ischemic Heart Disease Risk

Altogether, AB Science has been evaluating a tyrosine kinase inhibitor, masitinib, for a variety of conditions including amyotrophic lateral sclerosis (ALS), COVID-19, and mastocytosis. But according to ALS News Today,…

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The N-Lorem Foundation is Developing Treatments for Patients With Ultra-Rare Diseases
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The N-Lorem Foundation is Developing Treatments for Patients With Ultra-Rare Diseases

  A recent article in the Encinitas Advocate heralded the dedication of Stan Crooke, n-Lorem Foundation’s CEO and founder. The nonprofit Foundation’s team acts as middlemen, working with patients who…

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New $15M Effort to Expand ALS Research and Find Biomarkers
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New $15M Effort to Expand ALS Research and Find Biomarkers

Target ALS has recently launched a new research initiative, in which they will dedicate $15 million to amyotrophic lateral sclerosis (ALS) research, specifically the discovery of biomarkers. These millions will…

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Brainstorm Shares the Results of its Phase 3 Study of NurOwn to Treat ALS
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Brainstorm Shares the Results of its Phase 3 Study of NurOwn to Treat ALS

  According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…

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New Discovery Allows Researchers to Study Mechanisms in Neurodegenerative Diseases from Beginning of life
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New Discovery Allows Researchers to Study Mechanisms in Neurodegenerative Diseases from Beginning of life

Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…

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