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Scientific Advances Increase Potential For Drugs To Treat ALS

Executive Summary

New understanding of the spectrum of CNS disorders and rising interest from biotechs have revealed new targets, new biomarkers and new opportunities to treat the fast-acting and deadly neurodegenerative disease. Add to that an open stakeholder forum hosted by FDA Feb. 25 that could clarify regulatory and clinical expectations, and the tide may be turning on amyotrophic lateral sclerosis.

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ALS Patients Ask FDA To See Risk Their Way

An FDA panel got an earful on the review and regulation of drugs for amyotrophic lateral sclerosis Feb. 25 during a public hearing convened under the agency’s plan to involve patient networks in the regulatory process, particularly where it concerns lowering barriers to drug development for ALS.

ALS Organization Does CRO Work On The Side To Support Research

To raise funds for its own ALS research, ALS Therapy Development Institute offers services in gene expression, disease modeling and drug screening for pharmas that think their candidate might work against the orphan neurodegenerative disease. It’s a win-win for ASLTDI, which also sees the service as a way of creating ALS champions in big pharma.

Draft Common Data Elements Connect MS, Other Neurological Diseases

NIH’s National Institute of Neurologic Disease and Stroke coordinated a project to get experts to agree on a set of common data elements for use in studies of different aspects of multiple sclerosis.

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