Great news that Synapix Bio have secured £13.2m to fund clinical trials for H-ABC/Tubb4a!
Oxford biotech firm secures £13.2m to develop first treatment for rare, incurable, deadly disease. (synaptixbio.com)
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H-ABC stands for Hypomyelination with atrophy of the basal ganglia and cerebellum click below to find out more.
Across the world over 150 children have been diagnosed with H-abc. Here you can read some of their stories.
Doctors are currently working on a treatment programme for H-abc, click below to find out more about the path to treatment.
Children with H-abc need your help! find out more about how you can donate to this cause and help find a cure.
WE ARE STRONGER TOGETHER THAN WE ARE ALONE.
WITH YOUR HELP WE WILL FIND THE CURE
At this time, the team at CHOP is looking at gene therapy to treat H-ABC, as its ability to cure cellular-level diseases is promising. Once they’re able to determine the best approach for gene therapy, they can then get started on obtaining FDA approval and launching a clinical trial.
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