Posts by: Iqra Mumal

Iqra Mumal

October 18, 2018October 18, 2018

Sarepta and Lysogene Sign Licensing Deal for LYS-SAF302, Potential Gene Therapy for Sanfilippo Type A

News

Sarepta Therapeutics has signed a licensing agreement with Lysogene for the development of investigational gene therapy LYS-SAF302 to treat mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome ... Read more

August 31, 2018

High-dose Genistein Shows No Clinical Benefit for Treating Sanfilippo Children, European Study Shows

News

Results from a Phase 3 clinical trial evaluating the use of high-dose genistein in children with Sanfilippo syndrome shows that its use has no meaningful clinical benefit. Sanfilippo syndrome — ... Read more

August 23, 2018August 23, 2018

Implanting Genetically Corrected Stem Cells Partially Restores Enzyme Activity in Sanfilippo Mice, Study Finds

News

Brain transplantation of genetically corrected stem cells partially restored enzymatic activity in mice with Sanfilippo syndrome, according to UCLA researchers. Their study, “Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver ... Read more

July 10, 2018July 10, 2018

New Gene Therapy Candidate for Sanfilippo Type C Ready for Clinical Trial Stage

News

An investigative gene therapy to treat patients with Sanfilippo syndrome type C was developed at the University of Manchester in England, and Phoenix Nest, a biotech based in the U.S., ... Read more

May 8, 2018May 8, 2018

Sanfilippo Patient’s High Levels of Specific Amino Acid Was Due to B12 Deficiency, Case Report Contends

News

A patient with Sanfilippo syndrome and poor nutritional status developed high levels of homocysteine — an amino acid likely due to a functional deficiency of vitamin B12, according to ... Read more

April 12, 2018April 12, 2018

Modified Version of NAGLU Enzyme Restored Activity and Cleared Heparin Sulfate in Mouse Study

News

Injecting a modified version of the NAGLU enzyme directly into the brains of mice with Sanfilippo syndrome type B normalized the enzyme’s activity and cleared the accumulation of heparan ... Read more

February 15, 2018February 15, 2018

Abeona Reports Positive Data from Clinical Trial of ABO-102 for Sanfilippo Type A

News

Abeona Therapeutics announced that ABO-102 (AAV-SGSH), an investigational therapy for Sanfilippo syndrome type A (MPS IIIA), demonstrated significant and durable clinical effects at various time points in a clinical trial. Results ... Read more

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