Principal investigator and member of the consortium, Dr. Stephanie Cherqui said: gene therapy adds a functional copy of the defective gene, and delivers them to the appropriate cells of the body case of cystinosis, most. Damaged due. The absence of the CTNS gene. In other human diseases, have a person ‘s own stem cells already safe, and the stem cells could be be used multiple target tissues. For cystinosis patients, this strategy could be a reservoir of healthy stem cells in the bone marrow create for the life of the respond to respond to the progressive tissue damage of cystinosis, and traveling, to meet. Different organs the different organs of the patient. .
The consortium includes renowned researchers in stem cell and gene therapy, molecular biology and clinical pathophysiology. We are the goal the first stem cell and gene therapy clinical trial for cystinosis reality dedicated we, if all goes well, there is a clinical trial for a cure within the next three to four years, said consortium member Nancy Stack, a co – founder and trustee of the CRF. far-reaching implications for the application to other diseases with systemic defects similar to cystinosis – stack added that are the first published results also..The agency customary criteria for authorizing anticancer agent has been extension of life in general , however such guidance view real benefits drug as Avastin for some female, says the Journal. – The finality the life and death decisions taken by the consent of medications like Avastin principle a moral question, says the editorial office conclude that additional drug approvals still below to to FDA standards, to asked on on the judgments by dying patient (Wall Street Journal.. ‘Morally indefensible’spot Approval Standard modified In spite Avastin decision, tells Wall Street Journal editorial.
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