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Post-CHDI Huntington's Disease KOL Interview
March 16, 2015
Key Highlights
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- ISIS-SMNRx data for Spinal Muscular Atrophy shows brain penetration by an ASO and is a positive for the development of ASO HD-SNP for Huntington's Disease (ISIS).
- An AAV-1 construct presentation from Lisa Stanek showed amazing data where their group got widespread transfection of their virus upon stereotactic injection into the striatum with retrograde transport to the cortex (SNY).
- The AMARRYLIS trial of PF-02545920 targeting PDE10 is underway, and Omeros toxicity may be a positive for Pfizer if its compound-related and not mechanism-related (PFE).
- There has been some nice selectivity data for the ZFP TF - Huntingtin Repressor. Sangamo has selected a lead compound, they've done a toxicity study in a relevant model (SGMO, SHPG).
- There are still questions regarding the mechanism of PBT2 in Huntington's Disease (PRAN).
For our disclosures, please read the BioMedTracker Research Standards. BioMedTracker will be offering KOL Reports and Physician Pulse Surveys for purchase a la carte, or access to all reports and surveys can be purchased as a subscription to KOL Insight. For more information on KOL Insight subscription, please email BioMedTracker or call BioMedTracker Client Services at (858) 200-2357.
| Disease Group Covered: | Neurology |
| Indications Covered: |
Guillain-Barré Syndrome
Ischemic Stroke Post-Traumatic Stress Disorder (PTSD) Premenstrual Dysphoric Disorder (PMDD) Sleep Apnea Smoking Cessation Substance Use Disorder |
Additional Resources: