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New strategy for assessing natural history of leukodystrophies

New strategy for assessing natural history of leukodystrophies
medicalxpress.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from medicalxpress.com Daily Mail and Mail on Sunday newspapers.

United statesLaura adangLaura ann adangAdeline vanderverLeukodystrophy centerGlobal leukodystrophy initiative clinical trials networkDivision of neurologyMolecular genetics

New Approach to Evaluate Leukodystrophies Natural History Unveiled

New Approach to Evaluate Leukodystrophies Natural History Unveiled
miragenews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from miragenews.com Daily Mail and Mail on Sunday newspapers.

United statesAdeline vanderverLaura adangDivision of neurologyNational institutes of healthLeukodystrophy centerGlobal leukodystrophy initiative clinical trials networkMolecular geneticsNational institutesOnline march

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 .

United statesUnited kingdomDan williamsAdeline vanderverLeukodystrophy centerUniversity of utahDrug administrationRare paediatric disease

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.

United kingdomDan williamsAdeline vanderverOligonucleotides asosAnna colivicchiLeukodystrophy centreAntisense oligonucleotidesOxford business park

RARE Drug Development Symposium: Fostering Successful Connections Between Stakeholders to Accelerate Progress

RARE Drug Development Symposium: Fostering Successful Connections Between Stakeholders to Accelerate Progress
globalgenes.org - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from globalgenes.org Daily Mail and Mail on Sunday newspapers.

United statesYssa dewoodyAdeline vanderverIlene penn millerIlene millerRare disease clinical research networkAmerican epilepsy societyEpilepsy leadership council advocacy coCommon fund undiagnosed disease networkRare epilepsy network ex officioOffice of rare diseases researchEpilepsy research benchmarksFoundation cooperative agreementInternational cofounderCommission for neurodevelopmental cnvsData management coordinating center