Jos�, Ben & Life Today
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Great Results Through Understanding
Sat, Mar 18, 2017 -
'Right To Try' Congressional Bill for ALS Patients
Thu, Jul 14, 2016 -
SB Independent - Ice Bucket Challenge: One Year Later
Wed, Aug 19, 2015
The aim of this research is to characterize the relationships between clinical phenotype, genetic background, representation of phenotype in induced pluripotent stem cells and correlation to drug response in ALS patients.
These goals will be accomplished by obtaining a patient’s consent to collect tissue samples (blood) for DNA sequencing and molecular profiling (RNA and protein) and a skin biopsy to develop an induced pluripotent stem cell line (iPS) for banking and high content drug screening at ALS TDI.
Patients will be asked to wear accelerometers on their wrists and ankles for one week per month to collect unbiased movement data and compare to the ALS FRS questionnaire, a self reporting web based questionnaire to monitor disease progression.
A database will be developed to link a patient’s genetic data, molecular data, and high content screening data with disease progression assessment methodologies.
Full protocol can be seen here: