Could cTAP’s new Duchenne prognostic score change how DMD trials measure mobility risk?
Find out how cTAP’s Duchenne prognostic score could improve DMD trial design and loss of ambulation prediction.
Pharma & Biotech
Features & Analysis
Can non-viral gene therapies solve the safety problem holding back genetic medicine?
Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.
Pharma & Biotech
Can Sarepta Therapeutics prove RNA interference works in FSHD1 and DM1 with early Phase 1/2 data
Read why Sarepta Therapeutics’ first siRNA data in FSHD1 and DM1 could matter for rare disease drug development and investor sentiment.
Pharma & Biotech
Sarepta’s sirolimus gambit: Can enhanced immunosuppression rescue ELEVIDYS for non-ambulatory Duchenne patients?
Sarepta's ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
Pharma & Biotech
Can Sarepta’s ELEVIDYS redefine Duchenne therapy? Inside the 3-year EMBARK data
Sarepta’s gene therapy ELEVIDYS shows 3-year durability in Duchenne muscular dystrophy. Find out what it means for patients, payers, and global rollout.
Pharma & Biotech
Can ELEVIDYS meet its promise? Sarepta’s pivotal Duchenne gene therapy trial nears major milestone
Sarepta’s EMBARK 3-year readout could clarify ELEVIDYS’ long-term role in Duchenne care. Find out what’s at stake in this pivotal gene therapy trial.