Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory …

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced ...
The American Journal of Managed Care

Key Functional Milestones Affected by Disease Progression

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for early intervention. Natural disease progression when SMA is left untreated ...
The American Journal of Managed Care

Describing SMA Progression Over Time

Panelists discuss how spinal muscular atrophy (SMA) progresses over time, affecting motor function differently in older patients vs infants, with experts noting the shift from traditional ...
Seeking Alpha

Avidity Biosciences' Del-zota Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Trial in People Living ...

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and ...