Approvals· August 22, 2026 · 19 min read
Zilganersen's September 22 PDUFA: Alexander Disease ASO & Intrathecal Access
By Ran Chen#FDA Approval#NDA Approval#Priority Review
Topic
Antisense
Antisense oligonucleotide therapies, evidence, payer coverage, specialty pharmacy routing, and rare-disease access.
Specialty· August 14, 2026 · 20 min read
Spinal Muscular Atrophy Treatment Access Landscape: Spinraza, Itvisma, & Evrysdi
By Ran Chen#Rare Disease#Gene Therapy#Antisense
Pricing & Access· August 13, 2026 · 20 min read
Epilepsy Treatment Access (2026): From $0.02 Generics to Zorevunersen
By Ran Chen#Antiepileptic Drugs#Antisense#Rare Disease