Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome type A, or mucopolysaccharidosis IIIA (MPS IIIA), is a rare progressively debilitating genetic condition in which people cannot break down heparan sulfate, a type of large sugar molecule called glycosaminoglycans (also known as GAGs, or mucopolysaccharides). This is caused by a lack of an enzyme called sulfoglucosamine sulfohydrolase (SGSH).
“It's really important for industry and advocacy to work hand in hand in order for us to develop treatments that are meaningful in the real lives of patients and families. Working together, we can develop clinical trials that measure things with real world impact.”
Cara O’Neill, M.D. - Chief Science Officer and Co-Founder, Cure Sanfilippo
Denali’s scientific approach to Sanfilippo syndrome Type A
Denali is evaluating DNL126 in a clinical study. This treatment is proposed to work by using Enzyme Transport Vehicle (ETV) technology to carry SGSH across the blood-brain barrier (BBB) and to other parts of the body.
DNL126 is being studied in a Phase 1/2 study of children with MPS IIIA to evaluate the appropriate dose of DNL126 in addition to gathering important safety data. Specifically, the study will evaluate the safety and potential impact of DNL126 on the behavioral, cognitive, and physical aspects of MPS IIIA.
To learn more about this study, including details about who may be eligible, visit ClinicalTrials.gov
DNL126 is an investigational drug, its clinical profile has not been established, and it has not been approved by any global health authority.
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Advocacy Organizations
These organizations are committed to the needs of people and families affected by Sanfilippo syndrome. Their websites may offer you more information, resources, and guidance.
By clicking these links, you will leave the Denali Therapeutics website and be redirected to a third-party site. Denali is not responsible for, nor does it endorse, the content, terms and conditions of use or privacy practices of external websites.
Cure Sanfilippo Foundation architects and funds cutting-edge research to accelerate discovery of a treatment or cure for Sanfilippo Syndrome so parents can dream of their children’s futures again.
Team Sanfilippo’s mission is in bringing families together for support, while funding clinical research and trials that look promising for improving quality of life for Sanfilippo children, and hopefully one day a cure.
IMPSN is a global community of informed and empowered organizations, who promote access to medicines, new research, and programs that lead to better quality of lives for patients and those affected by these diseases.
MPS Society UK transforms lives through support, research and awareness. We provide professional support to individuals and families affected by MPS, Fabry and related lysosomal conditions in the UK.
The National MPS Society’s mission is to serve those impacted by MPS and mucolipidosis (ML). It supports research, provides families with invaluable resources, and works to increase public and professional awareness. Visit this site to see a list of country-specific organizations outside the US.
Courageous Parents Network is a non-profit organization that orients and empowers parents and others caring for children with serious medical conditions, by providing resources and tools that reflect the experience and perspective of other families and clinicians.
Denali values all MPS IIIA community members and organizations. Additional resources may be available.
For questions, please contact the Denali Patient Advocacy team at patients@dnli.com.