News
Sanfilippo Syndrome Clinical Care International Guidelines (10/31/2022) - These guidelines provide guidance on Sanfilippo Syndrome-specific care management and monitoring of disease-related changes. The contents of this document have been reviewed and approved by medical and scientific professionals in the field. The file can be viewed and downloaded by clicking here. A collaborative effort between Cure Sanfilippo Foundation and Sanfilippo Children’s Foundation initiated and led this project through completion, as well… Continue Reading
Events
Registration is open for ADVANCE 2022: Sanfilippo Community Conference! (6/28/2022) - A virtual conference on July 7-8, bringing together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance the work to help children with Sanfilippo syndrome. Registration is at no-cost.REGISTER TODAY here: https://hopin.com/events/advance2022/registration Parents and caregivers of children with Sanfilippo syndrome don’t often get to see the… Continue Reading
More News
- Dr. Michael Beck, Mainz GE, MPS-focused physician has passed (9/23/2022) - September , 2022: It is with great sadness that we share, Dr. Michael Beck, based in Mainz GE passed away… Continue Reading
- Passing of Patient/Advocate Erica Thiel (2/7/2022) - Our beloved friend Erica Thiel passed away recently on Saturday, January 8. Erica was born on March 5, 1983, and… Continue Reading
- Lysogene awarded Innovation Passport by the UK MHRA for the GM1-gangliosidosis program (12/20/2021) - Paris, France — 20 December 2021 at 08:00 am CET — Lysogene (FR0013233475 – LYS), a phase 3 gene therapy platform Company… Continue Reading
- 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… Continue Reading
More Events
- POMPE PATIENT MEETING, August 2022 (6/22/2022) - Happy summer to all! We first want to thank everyone for their patience and understanding as our team finalizes details… Continue Reading
- Lysosomal Disease Network annual meeting 2022 (5/10/2022) - The Lysosomal Disease Network’s annual “all consortium” meeting has been scheduled for June 24th, 2022, Noon - 4 PM EST.… Continue Reading
- Precision Metrics for Cognition (3/2/2022) - The National MPS Society is pleased to bring a special opportunity to our supporters who develop and administer clinical trials. This… Continue Reading
- WORLDSymposium Annual Meeting (12/29/2021) - WORLDSymposium annual meeting, February 6 - 11, 2022 at the Manchester Grand Hyatt, San Diego, CA Five full days of… Continue Reading
More 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 syndrome type A) by enzyme replacement therapy (ERT) and hematopoietic stem… Continue Reading
- Lasting Lessons from the Pandemic: Advancing the Understanding of Special Education and Therapeutic Needs of Children with Neurodegenerative Disorders – special white paper for review (8/23/2021) - Check out this important discussion: When the COVID-19 pandemic halted in-person instruction, educators, therapists, and parents faced unprecedented challenges as… Continue Reading
- Experts’ views on COVID-19 vaccination and the impact of the pandemic on patients with Gaucher disease (7/14/2021) - The current outbreak of the novel severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2), causing coronavirus disease 2019 (COVID-19), has become a… Continue Reading
- Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe Disease – new PubMed article! (5/12/2021) - Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe Disease.Thompson-Stone R, Ream MA, Gelb M, Matern D, Orsini… Continue Reading
