We are grateful for their willingness ... rare-disease research and treatment development." "We are entering a new era for LGMD," Knudson said. "Now we need policy to keep pace with the science." ... The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation ... We are grateful for their willingness to listen to patients and work across party lines to strengthen rare-disease research and treatment development." "We are entering a new era for LGMD," Knudson said. "Now we need policy to keep pace with the science." ... The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation for people living with limb-girdle muscular dystrophy (LGMD) and other neuromuscular rare diseases. /PRNewswire/ -- Advocates living with limb-girdle muscular dystrophy (LGMD) traveled from across the country to Washington this week, bringing their lived... Rare-disease development programs often operate with limited funding, data and eligible patient populations. When regulatory expectations are unclear, change late in development or are applied inconsistently, promising programs can stall and investment can disappear. The Speak Foundation Honors Congressional Champions · As part of LGMD Day on the Hill activities, The Speak Foundation presented its 2026 Congressional Champion for Limb-Girdle Muscular Dystrophy Awards to Rep. Advocates asked Congress to support policies that recognize both the scientific challenges of studying rare diseases and the expertise patients bring to the process. The Speak Foundation supports engaging patients before trials are designed so their perspectives can inform endpoints, eligibility criteria, visit schedules and outcomes that matter in daily life.

Original title: “Speak Foundation Brings LGMD Advocates Representing More Than 20 states to Capitol Hill, Honors Bipartisan Rare Disease Champions”

The FDA’s priority review acceptance of BridgeBio’s BBP-418 is another step toward what William Blair previously dubbed a “diversified commercial portfolio.” It also adds to the rapidly building momentum in muscular dystrophy more broadly.

Original title: “FDA accepts BridgeBio’s application for potential first limb-girdle muscular dystrophy drug”

limb girdle muscular dystrophy associated with trim32 variants a national cohort study

Original title: “Limb Girdle Muscular Dystrophy Associated With TRIM32 Variants: A National Cohort Study.”

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular dystrophy.

Original title: “BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug”