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What happens when a $2 million gene therapy is not enough

Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to. -Today at 05:00 am- MarketScreener

United statesBen kutschkeRoger hajjarRussell butterfieldEric coxElizabeth kutschkeJerry mendellSara ledwithAmanda cookMaha radhakrishnanSitra tauscher wisniewskiSteven pearsonRoche evrysdiStacie dusetzinaCaroline humerSree chaguturu

What happens when a $2 million gene therapy, once world s priciest drug, is not enough

Zolgensma has been given to more than 3,000 children globally

Oak parkUnited statesBen kutschkeAmanda cookSitra tauscher wisniewskiRussell butterfieldRoche evrysdiJerry mendellMaha radhakrishnanStacie dusetzinaElizabeth kutschkeSteven pearsonRoger hajjarSree chaguturuVasant narasimhanDrug administration

What happens when a $3m gene therapy is not enough

LOS ANGELES - Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to walk, talk, swallow or even breathe. So when in 2021 his parents heard about Zolgensma – a one-time therapy costing millions of dollars that.

United statesRussell butterfieldBen kutschkeStacie dusetzinaSitra tauscher wisniewskiJerry mendellRoger hajjarMaha radhakrishnanAmanda cookElizabeth kutschkeSteven pearsonRoche evrysdiSree chaguturuVasant narasimhanNashville vanderbilt university school of medicineDrug administration

What happens when a $2 million gene therapy is not enough?

What happens when a $2 million gene therapy is not enough?
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United statesElizabeth kutschkeStacie dusetzinaJerry mendellSteven pearsonBen kutschkeRoger hajjarRussell butterfieldSitra tauscher wisniewskiMaha radhakrishnanSree chaguturuVasant narasimhanAmanda cookRoche evrysdiInstitute for human data scienceUnitedhealth group

Realising milestones with gene therapy for SMA

Novartis Gene Therapies' Dr Sitra Tauscher-Wisniewski shares what shaped the success of one of the few commercially-available gene therapies.

Eli lillySitra tauscher wisniewskiAstellas pharmaHealth resourcesUniversity of viennaMuscular dystrophy association conferenceClinical development analytics at novartis gene therapiesNational institute for healthClinical development for novartis gene therapiesDrug administrationClinical development analytics of novartis gene therapiesNational institute of neurological disordersHead of clinical developmentClinical development analyticsGene therapie vice presidentClinical development