Sanfilippo disease

Biopotency signals demonstrated in Sanfilippo type A Gene therapy trial, provides Abeona Therapeutics

Sanfilippo type A Gene therapy trial shows biopotency signals, provides Abeona Therapeutics

2 August 2016 | By Abeona Therapeutics Inc

Abeona Therapeutics provides an update on its first Sanfilippo patient and reports that early biopotency signals suggest a reduction of heparan sulfate in the urine of patients and if confirmed will suggest that intravenous administration of ABO-102 may ultimately provide both central nervous system and systemic benefits to patients...

news

Stem cell gene therapy for Sanfilippo disease ready for human trial

11 May 2016 | By Victoria White, Digital Content Producer

University of Manchester and CMFT researchers are to work with Orchard Therapeutics to test a stem cell gene therapy for Sanfilippo disease in a human trial...

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Sanfilippo disease

Sanfilippo type A Gene therapy trial shows biopotency signals, provides Abeona Therapeutics

Stem cell gene therapy for Sanfilippo disease ready for human trial