March 30, 2021 – Roche Pharmaceuticals Suspends Huntington's Disease Trial

This disappointing result was unexpected based on preclinical studies undertaken by Ionis Pharmaceuticals who, along with the Center, has championed the use of antisense therapeutics for treating neurologic disorders. Click here for more information.

January 1, 2021 – Reference in Science Magazine

Research undertaken by Dr. Smith on an antisense oligonucleotide clinical trial, published in Lancet Neurology in 2013, was referenced in an article in Science titled "RNA-targeted drugs for neuromuscular diseases" (Vol. 371, Issue 6524, pp. 29-31).

December 2020 – Neurotherapeutics in the Era of Translational Medicine

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