News

Neurotherapeutics in the Era of Translational Medicine is an Elsevier publication edited by Richard A. Smith, Brian K. Kaspar, and Clive N. Svendsen. This anthology provides a comprehensive review on the progress of translational research in neurotherapeutics for neurologic disorders.

Table of Contents

  1. Development of a molecular therapy for the SOD1 familial variant of ALS

  2. Unraveling molecular biology of C9ORF72 repeat expansions in amyotrophic lateral sclerosis-frontotemporal dementia: Implications for therapy

  3. Translating molecular therapies for spinal muscular atrophy

  4. From uncovering the mechanism of transthyretin aggregation to the drug tafamidis for ameliorating neurodegeneration and cardiomyopathy

  5. A novel treatment for Parkinson's disease and ALS: Combined cell and gene therapies

  6. Parkinson's disease: Genetic-driven therapeutic approaches

  7. Neural stem cell-mediated brain tumor therapy

  8. Advances in migraine therapeutics: The role of calcitonin gene-related peptide

  9. The impact of translational research on the development of therapeutic agents for multiple sclerosis

  10. Advances in treating myasthenia gravis

  11. The endocannabinoid system as a target for the treatment of neurological disorders

  12. Identification of biomarkers for diagnosing and monitoring therapy in the treatment of neurologic disorders

  13. 3D Alzheimer's disease in a dish: Implications for drug discovery

  14. Improving clinical trial efficiency with machine learning models of disease progression

  15. Positron emission tomography imaging agents for evaluating the pathologic features of Alzheimer's disease and drug development

  16. Repurposing FDA-approved pharmaceuticals: The development of Nuedexta

  17. Bioethics in the era of translational medicine

  18. Government efforts to promote the development of neurotherapeutics for the treatment of neurological diseases

  19. Innovative funding models for neurotherapeutics


Dr. Smith and his colleagues (Dr. Cleveland, Dr. Ferguson, Dr. Bennett, and Dr. Miller) were the recipients of the Healey Center International Prize for Innovation in ALS, which was presented on December 6th at the 2019 International MND Symposium in Perth, Australia. The official news release by the Sean M. Healey and AMG Center for ALS can be found here.


The following are videos of the award presentation:

Merit Cudkowicz introducing the award.

Dr. Tim Miller's presentation.

Dr. Richard Smith's presentation.

Dr. Don Cleveland's presentation.



Left to right: Regan Asnes Healey; Don Cleveland, Ph.D.; Richard Smith, M.D.; Toby Ferguson, M.D., Ph.D.; Timothy Miller, M.D., Ph.D.; Merit Cudkowicz, M.D., MSc.


Photo courtesy of Sean M. Healey and AMG Center for ALS.

The results of a Phase 2 trial using a drug strategy pioneered by our director, Richard Smith M.D., in collaboration with Ionis Pharmaceuticals and the University of California, San Diego has been reported to demonstrate promising results in a Biogen-sponsored treatment trial. Click here for more information.