ALS Recent Progress Evaluation at MDA 2023.

Published Date: 01 Mar 2023

In the MDA session track on amyotrophic lateral sclerosis, Stanley Appel, MD, provided his perspective on the condition, the most recent scientific developments that will benefit patients, and the direction that research and treatment in the area are taking.

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AskBio noted that AB-1009 also recently received fast track and orphan drug designations from the FDA.

A phase 2a trial showed that NLX-112 was safe and well tolerated in patients with Parkinson disease, while also reducing levodopa-induced dyskinesia and motor symptoms, supporting further clinical development.

A Phase 1 study in healthy lactating women found that a single 10‑mg dose of zavegepant nasal spray was generally safe, well tolerated, and resulted in minimal infant exposure through breast milk.

IntraBio reported that levacetylleucine showed symptom improvement in the phase 3 IB1001-303 study for ataxia-telangiectasia, and it intends to proceed with regulatory submissions in multiple regions.

The chief medical officer at the Parkinson’s Foundation detailed how mood and behavioral symptoms shape quality of life in Parkinson disease and how clinicians can better identify and manage them.

The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.

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