Fragile X syndrome
FAQs
- Q1 What is Fragile X syndrome?
- Q2 What are the characteristics of Fragile X syndrome?
- Q3 What causes Fragile X syndrome?
- Q4 How does Fragile X syndrome impact on carers, friends and family?
- Q5 What are NPL-2005 and NPL-2009?
- Q6 Where and when will NPL-2005 and NPL-2009 be made available to patients?
- Q7 Can my child take part in clinical trials for NPL-2005 or NPL-2009?
Q5 What are NPL-2005 and NPL-2009?
These are Neuropharm’s separate but potentially complementary drug compounds and approaches to treating Fragile X syndrome. They are currently in our development pipeline.
- NPL-2005, seeks to target the behavioral symptoms of Fragile X syndrome. A Phase II clinical trial in children and adolescents at a major European university hospital has been completed. This was the first sponsor lead study of a pharmaceutical in Fragile X syndrome in Europe. The results of this open label study were presented in July 2008 at the Fragile X Foundation’s 11th International Fragile X Conference in St. Louis, Missouri.
- NPL-2009 is a targeted treatment directed at biological pathways that may underlie symptoms. In conjunction with FRAXA, Neuropharm has completed an exploratory Phase II trial of NPL-2009 in male and female adult patients with Fragile X syndrome. The results of this single dose, open label study which investigated tolerability and pharmacokinetics of NPL-2009 in this patient group were published in January 2009 in the peer-reviewed Journal of Medical Genetics. This was the world’s first clinical study using a targeted treatment for Fragile X syndrome.