A Guide to Becker Muscular Dystrophy (Patient Brochure)

Diagnosis and Management Guide for Families

Becker Muscular Dystrophy Clinical Trials

Becker Muscular Dystrophy Clinical Trials

Patient Advocacy

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Genetic Testing

Becker Education & Engagement Day

References

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  2. Kiriaev L, Baumann CW, Lindsay A. Eccentric contraction-induced strength loss in dystrophin-deficient muscle: preparations, protocols, and mechanisms. J Gen Physiol. 2023;155(2):e202213208. doi:10.1085/jgp.202213208
  3. Darras BT, Urion DK, Ghosh PS. Dystrophinopathies. In: Adam MP, Bick S, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, eds. GeneReviews®. University of Washington, Seattle; 2000.
  4. Magot A, Wahbi K, Leturcq F, Jaffre S, Péréon Y, Sole G; French BMD working group. Diagnosis and management of Becker muscular dystrophy: the French guidelines. J Neurol. 2023;270(10):4763-4781. doi:10.1007/s00415-023-11837-5
  5. Latimer R, Street N, Conway KC, et al; Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Secondary conditions among males with Duchenne or Becker muscular dystrophy. J Child Neurol. 2017;32(7):663-670. doi:10.1177/0883073817701368
  6. Bello L, Riguzzi P, Capece G, et al. Longitudinal changes of motor function in Becker muscular dystrophy. Neurol Genet. 2025;11(4):e200285. doi:10.1212/NXG.0000000000200285
  7. Aartsma-Rus A, Ginjaar IB, Bushby K. The importance of genetic diagnosis for Duchenne muscular dystrophy. J Med Genet. 2016;53(3):145-151. doi:10.1136/jmedgenet-2015-103387
  8. Niks E, Collins S, Byrne B. Becker muscular dystrophy: could altering the natural history of decline help tackle unmet medical need? Symposium presented at: 28th International Annual Congress of the World Muscle Society (WMS); October 8, 2023; Charleston, South Carolina.
  9. Finsterer J, Stöllberger C. Cardiac involvement in Becker muscular dystrophy. Can J Cardiol. 2008;24(10):786-792. doi:10.1016/s0828-282x(08)70686-x
  10. Bello L, Campadello P, Barp A, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439
  11. van de Velde NM, Hooijmans MT, Sardjoe Mishre ASD, et al. Selection approach to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522. doi:10.1212/WNL.0000000000012233
  12. De Wel B, Iterbeke L, Huysmans L, et al. Muscle MR imaging and clinical outcome measures in adults with Becker muscular dystrophy over 18-months follow-up. Poster presented at: 28th International Annual Congress of the World Muscle Society (WMS); October 6, 2023; Charleston, South Carolina.
  13. Schrama EJ, Koeks Z, van de Velde NM, et al. Sensitivity of different clinical outcome measures in assessing adults with Becker muscular dystrophy: a 3-year natural history study. Neurology. 2025;105(7):e214071. doi:10.1212/WNL.0000000000214071
  14. MacDougall J, James M, McDonald C, et al. North Star (NSAA and NSAD) functional assessments in individuals with Becker muscular dystrophy. Poster presented at: MDA Clinical and Scientific Conference; March 3-6, 2024; Orlando, Florida.
  15. Data on file. Edgewise Therapeutics.
  16. Great Ormond Street Hospital NHS Foundation Trust & The Newcastle upon Tyne Hospitals NHS Foundation Trust. North Star Ambulatory Assessment (NSAA) V 2.2. UK North Star Clinical Network and Muscular Dystrophy UK. September 30, 2020. Updated July 4, 2024.
  17. Punnoose AR, Burke AE, Golub RM. Muscular dystrophy. JAMA. 2011;306(22):2526. doi:10.1001/jama.2011.1794
  18. Bushby KMD, Gardner-Medwin D. The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy: I. natural history. J Neurol. 1993;240(2):98-104. doi:10.1007/BF00858725
  19. James MK, Alfano LN, Muni-Lofra R, et al. Validation of the North Star Assessment for limb-girdle type muscular dystrophies. Phys Ther. 2022;102(10):1-9. doi:10.1093/ptj/pzac113
  20. del Rocío Cruz Guzmán O, Chávez García AL, Rodríguez-Cruz M. Muscular dystrophies at different ages: metabolic and endocrine alterations. Int J Endocrinol. 2012;2012:485376. doi:10.1155/2012/485376
  21. Becker Muscular Dystrophy (BMD). Muscular Dystrophy Association (MDA). July 2025. Accessed August 22, 2026. https://www.mda.org/sites/default/files/2025/07/BMD-Fact-Sheet.pdf
  22. Riguzzi P, Borland H, James MK, et al. Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines. J Neurol. 2025;272(7):448. doi:10.1007/s00415-025-13126-9
  23. Ho R, Nguyen ML, Mather P. Cardiomyopathy in Becker muscular dystrophy: overview. World J Cardiol. 2016;8(6):356-361. doi:10.4330/wjc.v8.i6.356
  24. Del Rio-Pertuz G, Morataya C, Parmar K, Dubay S, Argueta-Sosa E. Dilated cardiomyopathy as the initial presentation of Becker muscular dystrophy: a systematic review of published cases. Orphanet J Rare Dis. 2022;17(1):194. doi:10.1186/s13023-022-02346-1
  25. De Wel B, Willaert S, Nadaj-Pakleza A, et al. Respiratory decline in adult patients with Becker muscular dystrophy: a longitudinal study. Neuromuscul Disord. 2021;31(3):174-182. doi:10.1016/j.nmd.2020.12.010
  26. Angelini C, Marozzo R, Pegoraro V. Current and emerging therapies in Becker muscular dystrophy (BMD). Acta Myol. 2019;38(3):172-179.
  27. Donovan J, Bronson A, Armstrong N, Martin A. Patient-reported aspects of Becker muscular dystrophy from The Duchenne Registry: a comprehensive registry for Duchenne and Becker muscular dystrophy (BMD). Poster presented at: 27th International Hybrid Annual Congress of the World Muscle Society; October 2022; Halifax, Nova Scotia.
  28. Abukmail E, Bakhit M, Hoffmann TC. Exploring individuals’ perceptions and acceptability of a “wait and see” approach for managing self-limiting illnesses: a qualitative study. Patient Educ Couns. 2024;118:108032. doi:10.1016/j.pec.2023.108032
  29. Babcock MA, Kostova FV, Moxley RT III, Chamberlain JS, Maria BL. Muscular dystrophy: new opportunities for diagnosis and treatment. J Child Neurol. 2010;25(9):1080-1097. doi:10.1177/0883073810371000
  30. Parent Project Muscular Dystrophy. New Diagnosis and Early Care Guide. ParentProjectMD.org. Accessed August 28, 2026. https://www.parentprojectmd.org/wp-content/uploads/2024/06/PPMD_NewDiagnosisBooklet-23_final_web.pdf
  31. American Academy of Neurology (AAN). American Academy of Neurology Muscular Dystrophy Quality Measurement Set. May 28, 2014. Accessed August 22, 2026. https://www.aan.com/siteassets/home-page/policy-and-guidelines/quality/quality-measures/13mdmeasureset_pg.pdf
  32. Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13. doi:10.1038/s41572-021-00248-3