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British biotech firm secures coveted FDA designation in bid to develop world's first treatment for rare, incurable and deadly disease - Vimarsana News

British biotech firm secures coveted FDA designation in bid to develop world's first treatment for rare, incurable and deadly disease

A leading UK biotech firm founded to develop the world’s first treatment for a rare, incurable and deadly disease has secured its first Food and Drug Administration (FDA) designation. SynaptixBio, which aims to tackle TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and ...

Oxford firm to lead the way to find treatment for rare genetic disorder - Vimarsana News

Oxford firm to lead the way to find treatment for rare genetic disorder

A BIOTECH firm in Oxford is hoping to develop the first treatment for a rare genetic disorder.

CHOP Family from Vermont Helps Raise Awareness for Leukodystrophy - Vimarsana News

CHOP Family from Vermont Helps Raise Awareness for Leukodystrophy

Adeline Vanderver, MD Beth Leduc wasn’t expecting her son Christian to be diagnosed with leukodystrophy. Now, she’s making sure other families in