Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study

[1] Deenen JCW(2015)The epidemiology of neuromuscular disorders: a comprehensive overview of the literature J Neuromuscul Dis 2 73-85

[2] Horlings CGC(2006)Limb-girdle muscular dystrophy in the United States J Neuropathol Exp Neurol 65 995-1003

[3] Verschuuren JJGM(2018)Genetic landscape and novel disease mechanisms from a largeLGMDcohort of 4656 patients Ann Clin Transl Neurol 5 1574-1587

[4] Verbeek ALM(2018)Nusinersen versus Sham control in later-onset spinal muscular atrophy N Engl J Med 378 625-635

[5] van Engelen BGM(2017)Nusinersen versus Sham control in infantile-onset spinal muscular atrophy N Engl J Med 377 1723-1732

[6] Moore SA(2017)Single-dose gene-replacement therapy for spinal muscular atrophy N Engl J Med 377 1713-1722

[7] Shilling CJ(2021)Spinal muscular atrophy: from approved therapies to future therapeutic targets for personalized medicine Cell Rep Med 2 100346-968

[8] Westra S(2023)Recent trends in Antisense therapies for Duchenne muscular dystrophy Pharmaceutics 15 778-297

[9] Wall C(2023)Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥ 4 to < 8 years) with Duchenne muscular dystrophy: 1-year interim results from Study SRP-9001-103 (ENDEAVOR) Ann Neurol 94 955-869

[10] Wicklund MP(2009)Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins Ann Neurol 66 290-638

[1] Deenen JCW(2015)The epidemiology of neuromuscular disorders: a comprehensive overview of the literature J Neuromuscul Dis 2 73-85

[2] Horlings CGC(2006)Limb-girdle muscular dystrophy in the United States J Neuropathol Exp Neurol 65 995-1003

[3] Verschuuren JJGM(2018)Genetic landscape and novel disease mechanisms from a largeLGMDcohort of 4656 patients Ann Clin Transl Neurol 5 1574-1587

[4] Verbeek ALM(2018)Nusinersen versus Sham control in later-onset spinal muscular atrophy N Engl J Med 378 625-635

[5] van Engelen BGM(2017)Nusinersen versus Sham control in infantile-onset spinal muscular atrophy N Engl J Med 377 1723-1732

[6] Moore SA(2017)Single-dose gene-replacement therapy for spinal muscular atrophy N Engl J Med 377 1713-1722

[7] Shilling CJ(2021)Spinal muscular atrophy: from approved therapies to future therapeutic targets for personalized medicine Cell Rep Med 2 100346-968

[8] Westra S(2023)Recent trends in Antisense therapies for Duchenne muscular dystrophy Pharmaceutics 15 778-297

[9] Wall C(2023)Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥ 4 to < 8 years) with Duchenne muscular dystrophy: 1-year interim results from Study SRP-9001-103 (ENDEAVOR) Ann Neurol 94 955-869

[10] Wicklund MP(2009)Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins Ann Neurol 66 290-638