Children like Zaharrah need your support this tax-time
FSHD muscular dystrophy is devastating, slowly destroying every skeletal muscle in your body. For children with the early onset form of the disease, it can progress quickly and be even more severe.
Zaharrah wanted to learn to dance. Now she is in a wheelchair.
FSHD continues to rob too many children of the ability to run and play or dance.
Many even struggle to swallow.
Zaharrah relies on a feeding tube and constant care and support from her family. Because her facial muscles are affected too, she is unable to smile or show any of the emotions bubbling away inside her. Making and keeping friends at school has been hard, but her family know how to read her. They know her cheeky backtalk and her dreams. They know she has a fighting spirit.
We want to give children like her a chance to have a childhood. A chance to dance and smile and make friends. A chance to fully live. Will you help fund this study and put us on track for clinical trials that could make it all possible for Zaharrah and others like her?
Please Donate Now
All donations up to $100,000 will be matched, thanks to one of our generous supporters. Please, contribute now and help us unlock this matched funding.
Make your tax-deductible donation before 30 June and help children like Zaharrah access clinical trials for FSHD in Australia.
We have an exciting opportunity to progress clinical trials for children with early onset FSHD muscular dystrophy, as well as help other children with muscle degeneration.
Professor Silvia Blemker at the University of Virginia is preparing a normative study with 150 unaffected children and teens to collect baseline information and better understand many unknowns around bone and muscle development.
This world-first study uses the amazing Springbok scan made possible by past support from FSHD Global supporters like you. AI analyses will bring together layered MRI scans, chemical markers, and other information on bone and muscle development in children to create a paediatric normative database.
This database is crucial. If we do not have this information, no clinical trial for FSHD will be possible for anyone under the age of 16.
Please donate to help us reach our target of $600,000 faster.
With this study, we can attract clinical trials from all over the world and make the best new treatments, that are showing promising results in adults, available to children as well.
No parent with FSHD wants to be cured before their child. Spare them this agonising choice and help treatments for FSHD reach those like Zaharrah who need it the most.
"My hope is they find a cure or a treatment. Something for the little ones first. Because they are the ones affected the most, and they are the ones who succumb easier. They deserve it." - Zaharrah's dad, Mark.
Please support our world-first database
Early-onset FSHD presents a unique challenge in children: muscles are changing due to both disease and normal growth at the same time. Without a way to separate these effects, promising therapies may appear ineffective or be misinterpreted in children, delaying access to treatment.
Professor Silvia Blemker plans to create a paediatric normative database containing MRI scans, 3D modelling, chemical and protein markers, genetic and epigentic information, and more from 50 children and teens over time. This establishes a standard baseline for all future research.
The information from this normative study and the resulting database will enable clinical trials for children with other types of muscular dystrophy and neuromuscular diseases. It will be the launchpad for studies into muscle regeneration and has the potential to benefit children with a range of problems, including cerebral palsy, or those immobilised by cancer treatment, injury, coma or other causes of prolonged bedrest. This study is the key to unlocking so much hope.