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Related Experiment Video

Updated: May 3, 2026

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
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Opportunities for improving therapy development in ALS.

Lucie Bruijn1, Merit Cudkowicz,

  • 1ALS Association , Washington, D.C.

Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration
|January 30, 2014
PubMed
Summary
This summary is machine-generated.

To improve amyotrophic lateral sclerosis (ALS) therapy development, future clinical trials should target specific patient subgroups with biomarkers and incorporate innovative designs with patient advocate involvement.

Keywords:
Amyotrophic lateral sclerosisbiomarkersclinical trialstherapy

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Area of Science:

  • Neurology
  • Clinical Trials
  • Biomarkers

Background:

  • Stakeholders convened in May 2013 to discuss challenges in amyotrophic lateral sclerosis (ALS) therapy development.
  • The meeting involved The ALS Association and The Northeast ALS Consortium (NEALS).

Purpose of the Study:

  • To summarize issues and propose new directions for improving ALS therapy development.
  • To provide recommendations for future clinical trial designs in ALS.

Main Methods:

  • A round-table discussion among stakeholders was convened.
  • Issues and potential new directions were identified and summarized.

Main Results:

  • Future phase II clinical trials in ALS should target specific patient subgroups based on likely involvement in pathogenesis.
  • Trials should include biomarkers to monitor clinical progression and pharmacodynamic engagement.
  • Innovations in trial structure, design, and patient advocate involvement are recommended.

Conclusions:

  • Targeted therapies with biomarkers are crucial for advancing ALS treatment.
  • Improving clinical trial design and patient engagement can enhance therapeutic development for ALS.