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Scenic Biotech Enters Cooperative Research & Development Agreement with the National Institutes of Health for Genetic Modifier-Based Treatment Approach for Niemann-Pick Type C Disease

CRADA combines Scenic Biotech's genetic modifier platform with NIH's patient registry to unlock genetic modifiers as a path to novel therapies for Niemann-Pick Type C disease Scenic Biotech, a pioneer ....

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Repurposed ALS drug could slow gradual brain cell loss in Niemann-Pick disease type C1

Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in Niemann-Pick disease type C1 (NPC1), a rare genetic disorder affecting children and adolescents, suggests a study in mice by scientists at the National Institutes of Health. ....

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Repurposed ALS drug shows promise in mouse model of rare childhood genetic disorder

Repurposed ALS drug shows promise in mouse model of rare childhood genetic disorder
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