New understanding of Sly Syndrome
The scientist who discovered “Sly Syndrome” nearly four decades ago and a team of colleagues at Saint Louis University are a step closer to finding an approach to treat the rare genetic disease.
View ArticleSanfilippo syndrome compromises blood-brain barrier, leads to brain damage
A study into the effects of Sanfilippo Syndrome type B (MPS III B) has found that the barrier responsible for protecting the brain from the entry of harmful blood-borne substances is structurally and...
View ArticleInvestigators receive NIH grant to help move gene therapy for MPS IIIB toward...
Investigators at Nationwide Children's have received a grant from the National Institutes of Health to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in...
View ArticleLYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease
LYSOGENE announces the authorization by AFSSAPS (the French Agency for the Safety of Health Products) and by the Ethical Review Board of SAF-301, its leading intra-cerebral gene therapy phase I/II...
View ArticleRenaissance in drug development for rare diseases
Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug...
View ArticleGene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal...
A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects...
View ArticleStudy highlights benefits of using multiple injections to treat Sanfilippo...
New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.
View ArticleAbeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo...
Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted...
View ArticleResearch finding opens door to potential treatment for MPS IIIB
MPS IIIB is a devastating and currently untreatable disease that causes progressive damage to the brain, leading to profound intellectual disability, dementia and death -- often before reaching adulthood.
View ArticleGene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists...
Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off...
View ArticleNew understanding of Sly Syndrome
The scientist who discovered “Sly Syndrome” nearly four decades ago and a team of colleagues at Saint Louis University are a step closer to finding an approach to treat the rare genetic disease.
View ArticleSanfilippo syndrome compromises blood-brain barrier, leads to brain damage
A study into the effects of Sanfilippo Syndrome type B (MPS III B) has found that the barrier responsible for protecting the brain from the entry of harmful blood-borne substances is structurally and...
View ArticleInvestigators receive NIH grant to help move gene therapy for MPS IIIB toward...
Investigators at Nationwide Children's have received a grant from the National Institutes of Health to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in...
View ArticleLYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease
LYSOGENE announces the authorization by AFSSAPS (the French Agency for the Safety of Health Products) and by the Ethical Review Board of SAF-301, its leading intra-cerebral gene therapy phase I/II...
View ArticleRenaissance in drug development for rare diseases
Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug...
View ArticleGene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal...
A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects...
View ArticleStudy highlights benefits of using multiple injections to treat Sanfilippo...
New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.
View ArticleAbeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo...
Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted...
View ArticleResearch finding opens door to potential treatment for MPS IIIB
MPS IIIB is a devastating and currently untreatable disease that causes progressive damage to the brain, leading to profound intellectual disability, dementia and death -- often before reaching adulthood.
View ArticleGene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists...
Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off...
View Article