Recent event:

 AGSD-UK Annual Conference,
Nottingham,
28-29 October 2017.

 

Sodium Valproate in McArdle disease: a phase II clinical trial - news updates

Read more about the study on the MDC web site.


 

16/06/2014 – Recruiting participants

Full Title: A phase II pilot study to explore treatment with Sodium valproate in Adults with McArdle Disease (Glycogen Storage Disease Type V, GSDV)

Status: Open to Recruitment
Sponsor: University College London
Funder: Muscular Dystrophy Campaign (with support from AGSD-UK)
Principal Investigator: Dr Ros Quinlivan
Patients recruited: Target 8

This is a pilot phase II clinical trial of sodium valproate in patients with McArdle disease (glycogen storage disease type V). The objective is to determine whether treatment with sodium valproate can cause expression of the brain isoform of glycogen phosphorylase in skeletal muscle in people with McArdle disease, who are unable to produce the muscle isoform of glycogen phosphorylase. Eight participants will be recruited from the United Kingdom.

For information on the status of recruitment please contact Dr Renata Scalco at r.scalco@ucl.ac.uk


 

02/05/2013 – AGSD-UK and MDC announce joint fund raising

The AGSD-UK and the MDC are working jointly to raise funds for year two of the pilot trial. AGSD-UK will mail a joint leaflet to all McArdle people in the UK and promote the fund raising on social media. They will work on joint efforts to attract grants from charitable foundations.


 

10/10/2011 – MDC announce award of grant

The Muscular Dystrophy Campaign (MDC) announced a significant grant for a McArdle Disease trial. AGSD-UK is also assisting with this trial, which will be the first ever ‘safety and effectiveness’ trial of a drug in McArdle Disease. This is early stage work and any possible treatment is still many years away, but is a very exciting development.