PatientSpotlight, by PanaceaIntelPatientSpotlight

Topic

Newborn screening

Coverage of newborn screening across PatientSpotlight - the developments worth watching, the access reality, and what the next twelve months may bring. 2 pieces on this topic across therapy areas, newest first within each collection.

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Signals

2
SignalApr 29, 2026FDA

FDA approves leucovorin calcium as first treatment for CFD-FOLR1

The FDA has approved expanded use of Wellcovorin (leucovorin calcium) tablets for cerebral folate deficiency caused by confirmed FOLR1 gene variants, marking the first approved treatment for this rare neurological condition in adults and children.

RegulatoryGeneticsDiagnosisAccess
SignalApr 26, 2026FDA · regulatory-body · peer-reviewed

Newborn screening expansion is uneven across US states

RUSP additions tell only half the story - state-level implementation timelines stretch the diagnostic-to-treatment gap, and the variation has consequences for treatment-eligibility windows in time-sensitive rare disease.

DiagnosisNewborn screeningAccessRegulatory