Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)

2021 ◽  
Vol 132 ◽  
pp. S133-S134
Author(s):  
Kevin Flanigan ◽  
Nicholas Smith ◽  
Maria Couce ◽  
Maria Escolar ◽  
Kristen Truxal ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Single Dose ◽  
Phase 1 ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome

Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)

2021 ◽  
Vol 132 ◽  
pp. S139
Author(s):  
Maria de Castro ◽  
Kevin Flanigan ◽  
Bénédicte Héron ◽  
Nicole Muschol ◽  
Maria Couce ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Single Dose ◽  
Phase 1 ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome

Design and rational of the natural history study in preparation for the pivotal clinical trial for the treatment of Sanfilippo syndrome type A

2017 ◽  
Vol 120 (1-2) ◽  
pp. S25-S26
Author(s):  
Soraya Bekkali ◽  
Samantha Parker ◽  
Anupam Chakrapani ◽  
Roberto Giugliani ◽  
Benedicte Heron ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Natural History ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Natural History Study ◽  
Pivotal Clinical Trial

Cardiac Ultrasound Findings in Sanfilippo Syndrome Type A

2012 ◽  
Vol 105 (2) ◽  
pp. S34
Author(s):  
Brandon Hays ◽  
Elizabeth Braunlin ◽  
James Berry ◽  
Brenda Diethelm-Okita ◽  
Chester Whitley
Keyword(s):  
Type A ◽  
Cardiac Ultrasound ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Ultrasound Findings

Genotype, Disease Onset, And Neurocognitive Phenotype Of Sanfilippo Syndrome Type A

2012 ◽  
Vol 105 (2) ◽  
pp. S58 ◽  
Author(s):  
Chester B. Whitley ◽  
Renee Cooksley ◽  
Brenda Diethelm-Okita ◽  
Kathleen Delaney ◽  
Patrick Haslett ◽  
...  
Keyword(s):  
Disease Onset ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome

Mutation and haplotype analyses in 26 Spanish Sanfilippo syndrome type A patients: Possible single origin for 1091delC mutation

2001 ◽  
Vol 100 (3) ◽  
pp. 223-228 ◽  
Author(s):  
Amparo Chab�s ◽  
Magda Montfort ◽  
Maruxa Mart�nez-Campos ◽  
Anna D�az ◽  
Maria Josep Coll ◽  
...  
Keyword(s):  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Single Origin
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