News

Gaucher Awareness Video Premiere (8/31/2021) - The Gaucher Community Alliance is premiering its new video campaign to raise awareness about Gaucher disease during October’s Gaucher Awareness Month. The video shows the world the many ages, faces, ethnicities, languages and cultures it affects and will help medical professionals and people affected by Gaucher recognize symptoms. Join us for the premiere online, hosted… Continue Reading

Events

Letter from Sue Kahn, Executive Director, NTSAD: Starting a new chapter after stepping down from leadership (6/7/2021) - National Tay-Sachs & Allied Diseases AssociationJune 2021   Dear NTSAD Community,    It's with mixed emotions that I share with you that after nearly 14 years, I am stepping down from my role as NTSAD’s executive director at the end of October. I am looking forward to starting a new chapter in my life, knowing that NTSAD remains strong. I will… Continue Reading

Research

Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy (10/13/2021) - Treatments for Mucopolysaccharidosis IVA (MPS IVA, also called Morquio A syndrome) by enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) are limited in efficacy depending on the age of initiation and clinical phenotype. Thus, this study aims to assess the effects of treatments on MPS IVA patients compared to untreated MPS IVA patients… Continue Reading

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