Therapeutic Areas

Our expertise in developing effective treatments enables us to address complex challenges in key Therapeutic Areas.

Our core mission is to advance healthcare by creating and delivering highly effective treatments across various critical therapeutic areas. We are dedicated to pushing the boundaries of medical science through our robust research and development processes, resulting in groundbreaking solutions. By deeply understanding the unique needs of patients and healthcare providers worldwide in every therapeutic area, we tailor our methodologies to tackle the challenges at hand effectively. Our unwavering commitment to excellence places us at the forefront of medical progress, leading to positive outcomes and an improved quality of life for individuals. With a steadfast focus on innovation and patient well-being, we are driven to shape the future of medicine for generations to come.

Specialty & Consumer Health

Specialty &
Consumer Health

Duchenne Muscular Dystrophy - DMD - Italfarmaco SpA

Rare
Diseases

GB-ITF-26-000xx Date of preparation: August 2026

References

  1. Ryder S, Leadley RM, Armstrong N, Westwood M, de Kock S, Butt T, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis. 2017;12(1):79.
  2. Matthew Brooke et al. Collection of real-world data for Duchenne Muscular Dystrophy patients through a national registry: description of the current cohort in the UK. Poster P608 presented at World Muscle Society Annual Congress, 2024, Prague, Czech Republic.
  3. Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71(3):304-13.
  4. Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-67.
  5. Theret M, Rossi FMV, Contreras O. Evolving Roles of Muscle-Resident Fibro-Adipogenic Progenitors in Health, Regeneration, Neuromuscular Disorders, and Aging. Front Physiol. 2021;12:673404.
  6. Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484-6517.
  7. Lemos DR, Babaeijandaghi F, Low M, Chang CK, L ee ST, Fiore D, et al. Nilotinib reduces muscle fibrosis in chronic muscle injury by promoting TNF-mediated apoptosis of fibro/ adipogenic progenitors. Nat Med. 2015;21(7):786-94.
  8. Chapman MA, Mukund K, Subramaniam S, Brenner D, Lieber RL. Three distinct cell populations express extracellular matrix proteins and increase in number during skeletal muscle fibrosis. Am J Physiol Cell Physiol. 2017;312(2):C131–43.