Trusted Resources: News & Meetings
Latest announcements and gatherings
Advocacy Groups Unveil ‘Roadmap’ for Sanfilippo Research, Care
A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome Therapies, in an effort to accelerate research and develop effective therapies and care for families affected by Sanfilippo syndrome.
“The Roadmap aims to capture the historical landscape of research and clinical trials, the gaps that we currently see which are limiting progress, and ways in which we can move forward to address them collaboratively,” Cara O’Neill, MD, said in a press release. O’Neill, the chief science officer at Cure Sanfilippo Foundation, is a co-developer of the strategy plan.
Source: https://sanfilipponews.com/2022/02/24/advocacy-groups-unveil-roadmap-sanfilippo-research-care/
BookmarkRelated Content
-
Long-Term Effect of Hematopoietic Cell Transplantation on Systemic Inflammation in Patients With Mucopolysaccharidos...Mucopolysaccharidoses (MPS) are devastat...
-
Elosulfase Alfa in the Treatment of Mucopolysaccharidosis Type IVA: Insights From the First Managed Access AgreementManaged access agreements provide a cruc...
-
Gene Therapy for MPShttps://www.youtube.com/watch?v=Dh5pFgZd...
-
Johns Hopkins – Lysosomal Storage Disease CenterLysosomes are packets of enzymes or othe...
-
Mucopolysaccharidoses Diagnosis in the Era of Enzyme Replacement Therapy in EgyptUndegraded glycosaminoglycans (GAGs) ind...
-
Mucopolysaccharidosis Type III – Sanfilippo SyndromeMucopolysaccharidosis type III (MPS III)...
-
York Hob Moor Oaks School Boy Battles Hunter SyndromeThe family of a nine-year-old York boy a...
Powered by
