Dear sir I have problem Mascular Dystrophy with My sister His age is 13 years And he falls down 15 t0 20 times in a day And his mind as compare as 4 years child And his foots and hands looking as folding Please help us My sister's life getting destroyed day by day fastly Regards Sheeraz seelro From Pakistan Sindh Tell=+923322027622 ... See MoreSee Less
PTC Therapeutics have announced that they will be conducting a phase II clinical trial testing Translarna in 12 boys aged between 2-4 years who have Duchenne muscular dystrophy caused by a nonsense mutation. Unfortunately the study is only going to be held at centres in the US.
Translarna currently has conditional approval in Europe for treating boys aged 5 and over; this small-scale study marks the first step in the process of broadening this licence to include younger boys.
The pharmaceutical company, Acceleron, recently announced the results of its phase I clinical trial testing ACE-083 in healthy people. ACE-083 is an investigational protein therapeutic that binds and ...
“Muscle-wasting conditions can be extremely difficult to explain. These fantastic factsheets provide valuable, easy to understand and vital information about the myriad forms of Spinal muscular atrophy. They are a great resource for patients, parents, carers and medical professionals alike.” - James Lee who has spinal muscular atrophy.
Muscular Dystrophy and SMA Support UK have produced a new factsheet on SMA type 4 which is now available. >> qoo.ly/a39ps. ... See MoreSee Less
MDUK is delighted at the news that friend and supporter of the charity Sam Allardyce has been appointed England manager. Sam represented MDUK at the BGC charity fundraising day last September, and proved a big hit with supporters and families. We wish him the very best of luck in his new role! ... See MoreSee Less
What’s your view on newborn screening for Duchenne? Login and join our online discussion with Professor Francesco Muntoni this Monday, 2pm to 3.30pm. We’ll also be joined by Rachel Salmon, former newborn screening nurse in Wales, and families whose children were diagnosed via newborn screening, including Jeanette George (pictured with her son, Alex). >> qoo.ly/92u9r. ... See MoreSee Less