Rare Disease Day 2026: From Awareness to Access in a Volatile Year for Patients and Specialized Therapies

It’s not just about awareness but also a call to action for patients, families, employers, payers and policymakers to modernize the systems that shape access to care. It must focus on connecting innovation with the infrastructure that governs approval, coverage and affordability. The post Rare Disease Day 2026: From Awareness to Access in a Volatile…

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Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval

Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available…

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