Two First-Ever Rare Disease Approvals Landed the Same Day. Both Were Measured by Proxy.
Pasatru’s flare benefit is clinician-assessed only. GENGLYCOS is indicated to reduce cornstarch intake. Two firsts, both measured a step from the patient.
Gene Therapy
NeuroPulse Wk 26: Cockayne Gene Therapy World-First
NeuroPulse — Wk 26 · 2026 · Synopulse NeuroPulseDeep pine banner. An indigo neural network of neurons and synapses fills the right side, with a gold signal pulse firing along a chain of neurons, beside the NeuroPulse wordmark under the Synopulse and The Pulse kicker. Synopulse · The Pulse NeuroPulse This week in neuroscience Week … Read more