1202, 2017


Awareness is key. On 28 February 2017, the tenth year of Rare Disease Day will see thousands of people from all over the world come together to advocate for greater research into rare diseases. Rare Disease Day has, and continues to collaborate with organisations globally in order to put on [...]

111, 2016


Facio Therapies Announcement Facio Therapies announced today that oral treatment with one of Facio’s lead candidates results in significant reduction of the human muscle-toxic DUX4 protein in mice engrafted with human FSHD-affected muscle cells. Facio is the first in the FSHD field to achieve proof of principle in an [...]

3110, 2016


A major issue for FSHD FSHD is a condition that affects the function of skeletal muscles. However, this description does not include the myriad of other symptoms that people with FSHD are forced to deal with as the condition progresses. Fatigue is one of these symptoms. Fatigue is defined [...]

1310, 2016


How to tell the good from the not so good. There are literally thousands of scientific articles published every year around the world by research groups. Inevitably some will be great and others will not. Even a trained scientist can find interpreting research difficult especially with so many studies [...]

2109, 2016


Facio-therapies recently announced a breakthrough development in FSHD drug discovery. What is it? Facio-therapies have developed a high through-put screening platform that uses muscle cells from people with FSHD and actually measures DUX4 protein levels. Why is it a breakthrough? Most screening platforms use cells that are not specific [...]

3008, 2016


The Latest Study The role of exercise in muscular dystrophy has long been controversial and advice has oscillated from promoting exercise to counselling people with muscular dystrophy to avoid it entirely. A recent review of the available evidence concluded that exercise was not harmful for people with FSHD, but was unable [...]

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