Oct 20152015 MYOTUBULAR TRUST GRANT AWARDS ANNOUNCEDThanks to the support of our families, friends and community, the Myotubular Trust is delighted to announce two new grant awards...read more
Sep 2015ORPHAN DRUG STATUSAudentes Therapeutics receive orphan drug designation for AAV001 for the treatment of X-Linked Myotubular Myopathy in the US and in Europe...read more
Aug 2015A VERY SUCCESSFUL GOLF TOURNAMENTWe are running out of ways in which to thank Dave Blunsdon and Karen Vardy for organising the annual Jack Blunsdon Memorial Golf Trophy in memory of their son, Jack...read more
Aug 2015 2015 London 10k Runanother great turn out for the 10th Myotubular Trust 10k run read more
July 2015 BOB’S WRITING SKILLS RAISE A BOB OR TWOSale proceeds of ‘A Short History of The City of Leicester College’ written by our supporter, Bob Childs, raise a significant amount of money for the Trust read more
May 2015 2015 Hope Walk WeekendThis year saw four Hope Walks being held in Brighton, Cromer, London and Oldham, and a Walk Down a Tower too. Thank you to our wonderful supporters for arranging and taking part in these walks. read more
15th May 2015 Outstanding Award Dr Ana Buj Bello, Myotubular Trust grant recipient, receives Outstanding New Investigator Award from the American Society of Gene & Cell Therapy. read more
Myotubular Myopathy is a rare muscle condition which affects mostly boys at birth, and sometimes an even rarer form develops later in life affecting both boys and girls.
It causes profound muscle weakness. Most critically it affects the muscles that control breathing and swallowing and is a constant threat to life - even a simple cold could prove fatal. Sadly many do not survive their first year of life*.
Like all rare diseases it is very difficult to attract research funding for Myotubular Myopathy, despite the severity of its impact on the children affected. The Myotubular Trust has been set up to raise funds and begin the process of finding a cure.
The Myotubular Trust constantly aims for 100% of all funds raised by our supporters to go directly towards research, to help find a cure or treatment for children with myotubular myopathy**.
Orphan Drug Status
Audentes Therapeutics receive orphan drug designation for AAV001 for the treatment of X-Linked Myotubular Myopathy in the US and in Europe - read full story here
*source: Orphanet Journal of Rare Diseases. Centronuclear (myotubular) myopathy"
** achieved every year to date and to latest accounts at 31 December 2012