På den här sidan försöker vi att samla den information vi hittar om den forskning som pågår för Sanfilippos sjukdom. Framförallt för typ C (MPS IIIC)

Videon nedan förklarar Geneterapi för ärftliga metabola sjukdomar och är producerad av patientorganisationen VKS i Nederländerna.

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

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., has signed a licensing agreement to take the therapy to the clinical trial stage.

Royal Manchester Children’s Hospital

A two-year old patient at the Royal Manchester Children’s Hospital (RMCH) is the first in the world to receive a pioneering new ex vivo gene therapy treatment using lentiviral vector-modified autologous stem cells for the rare and life-limiting genetic condition, Mucopolysaccharidosis IIIA (MPSIIIA).

The gene therapy revolution is here

A couple of weeks back, you might have picked up a headline alerting us to the most expensive drug in history—a one off gene therapy cure for spinal muscular atrophy. Novartis have priced the drug Zolgensma at A$3 million (US$2.1 million).