Trusted Resources: News & Meetings
Latest announcements and gatherings
Sanfilippo Ideal for FDA’s Accelerated Approval Pathway, Foundation Says
The Cure Sanfilippo Foundation is encouraging the U.S. Food & Drug Administration (FDA) to consider Sanfilippo syndrome as a candidate for the agency’s accelerated approval pathway. According to a recent foundation-led study, families of Sanfilippo children are willing to accept modest treatment benefits even if they come with risks.
“Doing nothing is the biggest risk in Sanfilippo!” Glenn O’Neill, foundation president and father to a Sanfilippo child, said in a press release. His statement came at the end of a presentation Oct. 20 at the 13th Annual Rare Disease Scientific Workshop.
The FDA’s Accelerated Approval Program allows for earlier approval of therapies that treat serious conditions and fill an unmet medical need. Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a rare genetic disorder characterized by the mutations in genes that encode enzymes that break down the complex sugar molecule known as heparan sulfate.
BookmarkRelated Content
-
Growth Patterns in Children With Mucopolysaccharidosis Type I-Hurler After Hematopoietic Stem Cell Transplantation: ...The impact of hematopoietic stem cell tr...
-
I Want to Live in the Moment While There’s Still TimeMy daughter Abby has Sanfilippo syndrome...
-
A Guide to Understanding MPS IV (Morquio Syndrome)Mucopolysaccharidosis IV (MPS IV) is a r...
-
EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)JCR Pharmaceuticals Co., Ltd. announced ...
-
Gastrointestinal Manifestations in Mucopolysaccharidosis Type III: Review of Death Certificates and the LiteratureMucopolysaccharidosis type III (MPS III,...
-
Global Epidemiology of Mucopolysaccharidosis Type III (Sanfilippo Syndrome): An Updated Systematic Review and Meta-A...Mucopolysaccharidosis III, an autosomal ...
-
Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo AThe European Commission has granted an o...
Powered by
