Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Seelos Therapeutics‘ SLS-005 (trehalose) to treat Sanfilippo syndrome, the company has announced. The designation is granted to product applications for diseases that affect fewer than 200,000 children, 18 and younger, in the U.S. and provides certain…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Seelos Therapeutics‘ SLS-005 (trehalose) to treat Sanfilippo syndrome, the company has announced. Orphan drug designation aims to encourage the development of therapies for rare and serious diseases, through benefits such as seven years of market…

Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene therapy OTL-201 for Sanfilippo syndrome type A. OTL-201’s safety, tolerability, and efficacy is being investigated in the open-label, Phase 1/2 study (NCT04201405) taking place at Manchester University NHS…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Screening for metabolic disorders, such as Sanfilippo syndrome, should be considered in patients initially diagnosed with Landau Kleffner syndrome, according to a recent case study. The report, “An Uncommon Presentation of Mucopolysaccharidosis Type IIIb,” was published in the Iranian Journal of Child Neurology. MPS IIIB is…

Accumulation of the protein alpha-synuclein in the brain — a hallmark of neurodegenerative diseases like Parkinson’s — does not factor in the early disease course of Sanfilippo syndrome type A, a mouse study suggests. The study, “Early disease course is unaltered in MPS IIIA mice lacking α-synuclein,” was…

Researchers at the National Institutes of Health have developed an induced pluripotent stem cell (iPSC) line from a 1-year-old child with a mutated NAGLU gene, the gene responsible for Sanfilippo syndrome type B. These cells may now be used to generate other mature cells, such as neurons, to investigate…

Pentosan polysulfate sodium, an approved medication commonly used to treat bladder pain, will now be repurposed to potentially treat mucopolysaccharidosis (MPS) under the terms of two new licensing agreements. The oral therapy is approved by the U.S. Food and Drug Administration and marketed under the brand names Elmiron and Thrombocid, among others.

Phoenix Nest has acquired the exclusive rights from the University of Manchester for the development of a gene therapy to treat Sanfilippo syndrome type C. The company is planning to apply for its first in-human clinical trial with the U.S. Food and Drug Administration. The gene therapy uses…

A group of experts has discussed and released a series of recommendations for future trials’ design in Sanfilippo syndrome. The study, “Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III” was published in the journal Orphanet Journal of Rare Diseases. Sanfilippo syndrome, or mucopolysaccharidosis type III, belongs…