Survodutide — Indexed Studies

Delphy study on epidemiology, clinical management, disease burden, and treatment in paediatric patients with Duchenne muscular dystrophy in Spain.

Pitarch-Castellano I, Ortez-González CI, Nascimento-Osorio A et al.

Conformable Microelectrode Arrays Integrated with a Scoop-Shaped Slide-Well for Dynamic Electrophysiological Profiling of Patient-Derived Cardiac Organoids.

Kim YS, Jeong J, Heo G et al.

Metabolic Complications and Ultrasound-Detected Liver Abnormalities in Paediatric Patients With Duchenne Muscular Dystrophy.

Śledzińska K, Polat K, Kuchta A et al.

Long-read sequencing identifies complex structural variants in DMD patients.

Xie Y, Bao L, Yu X et al.

The Development of Spinal Deformity in Patients with Duchenne Muscular Dystrophy: Clinical Assessment, Surgical Considerations and Recommendations for Treatment.

Tsirikos AI, Roberts SB

From case to caution: hyponatremia in a patient with Duchenne muscular dystrophy on vamorolone and lessons for clinicians.

Murphy C, Nasomyont N, Tian C et al.

Assessing biomarkers of bone metabolism and the role of the interleukin-6 signaling pathway in patients with Duchenne muscular dystrophy.

Guridi M, De Ford C, See CG et al.

Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil.

Roesch EW, Colpani V, Dória GAA et al.

Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041.

Wu S, Vlodavets D, de Queiroz Campos Araujo AP et al.

Unmet Needs in the Care of Patients with Duchenne Muscular Dystrophy in Brazil.

Araujo APQC, Barbosa AVS, Becker MM et al.

Optical Genome Mapping and Long-Read Sequencing Identifies a Novel Dystrophin Gene Inversion in a Patient With Duchenne Muscular Dystrophy.

Gallagher R, Aguilera A, Villarreal SA et al.

Investigation of Walking Ability in 161 Patients With Duchenne Muscular Dystrophy.

Guo Y, Wu X, Xie Y et al.

Thorough QT Study on the Effect of Therapeutic and Supratherapeutic Dosing of Givinostat in Healthy Volunteers.

Mercuri E, Byrne B, Willis T et al.

Euglycemic ketoacidosis in a nondiabetic patient with Duchenne muscular dystrophy on dapagliflozin: comment.

Stöllberger C, Finsterer J

Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy.

Leon-Astudillo C, Salabarria SM, Chadwick CB et al.

Co-occurring DMD, GJA1, and novel FYCO1 variants in a proband from a consanguineous oculodentodigital dysplasia family: a rare multi-locus case report.

Abhishek K, Mohanta NK, John J et al.

Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy.

Lochmüller H, Gonorazky H, Nigro E et al.

Randomised trial of Aureobasidium pullulans-produced beta 1,3-1,6-glucans in patients with Duchenne muscular dystrophy: favourable changes in gut microbiota and clinical outcomes indicating their potential in epigenetic manipulation.

Raghavan K, Dedeepiya VD, Yamamoto N et al.

Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study.

Muntoni F, Nascimento A, Shin J et al.

Safety and Efficacy of Tamoxifen in Patients with Duchenne muscular dystrophy: open Label Extension of TAMDMD Trial.

Zwingli G, Putananickal N, Schmidt S et al.

Motor Function Changes in Duchenne Muscular Dystrophy: A Case Series Using Conventional and Spinal Muscular Atrophy-Based Assessments During Viltolarsen Treatment.

Iwayama H, Numoto S, Azuma Y et al.

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.

Shieh PB, Proud C, Diamond T et al.

Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes.

Malandrini F, Spataro C, Meregaglia M et al.

Prognostic value of right ventricular-pulmonary artery coupling in patients with muscular dystrophies.

Fayssoil A, Mansencal N, Chaffaut C et al.

Patient-Derived 3D Bioprinted Cardiac Organoid Constructs Reveal Key Pathological Features of Duchenne Muscular Dystrophy.

Marini V, Campaner Socias M, Dimopoulos A et al.

Lumbopelvic stabilization-based physiotherapy and rehabilitation and urotherapy for lower urinary tract dysfunction in Duchenne Muscular Dystrophy: a randomized controlled trial.

Öztürk D, Ayşe Karaduman A, Akbayrak T

Refining functional phenotypes in an international cohort of untreated paediatric type 2 and 3 SMA patients using the Revised Hammersmith Scale.

Milev E, Stimpson G, Ramsey D et al.

Symptomatic Pneumoperitoneum After Percutaneous Radiological Gastrostomy in Patient With Duchenne Disease Dependent on Non-Invasive Mechanical Ventilation.

Lozano Cuesta P, Ruiz Álvarez I, Guerassimova I et al.

Effectiveness of aquatic therapy on balance and functionality in children with Duchenne and Becker muscular dystrophy : a prospective controlled pilot study.

Yılmaz CU, Karabulut SS, Forestier FBE et al.

Evaluation of repolarization abnormalities with 12-lead ECG and 24-hour Holter ECG monitoring in patients with Duchenne muscular dystrophy.

Didinmez Taşkırdı E, Ünalp A, Yılmaz Ü et al.

A Rare Co-occurrence of Duchenne Muscular Dystrophy and Glycerol Kinase Deficiency Associated With Xp21 Contiguous Gene Deletion Syndrome: A Case Report.

Marques L, Lipari Pinto P, Coelho J et al.

Systematic analysis of the adverse effects of used clinical antisense oligonucleotide drugs in DMD patients based on the FAERS database.

Zhang XF, Hu WG, Hu J

Regenerative Index reveals declining muscle regeneration in paediatric patients with Duchenne muscular dystrophy.

Smid JK, McPherson CA, Monast JG et al.

Genotype-Dependent Morpho-Mechanical Profiling of Patient-Derived Human Myotubes on Nanogrooved Substrates.

Crépin R, Aït Ouailal A, Joanne P et al.

Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.

Mendell JR, Muntoni F, McDonald CM et al.

A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions.

Wang T, Daoud C, Dubois A et al.

Variant burden and severity of cardiomyopathy in patients with DMD-related Duchenne muscular dystrophy.

Geddes GC, Ware SM, Schwantes-An TH et al.

Epidemiology, treatment practices, and societal and economic burden of Duchenne muscular dystrophy in Finland: A retrospective register and chart review study.

Isohanni P, Auranen M, Vesikansa A et al.

A natural history study of Chinese individuals with Duchenne muscular dystrophy: Results from 2 years of follow-up and beyond.

Li X, Yang X, Chang X et al.

Dual-task performance as a clinical marker of upper limb decline in Duchenne muscular dystrophy.

Savas-Kalender D, Kurt-Aydin M, Tarsuslu T et al.

Survey of Adrenal Insufficiency Management for Duchenne muscular dystrophy in Italy.

Tornese G, Wong SC, Sbrocchi AM et al.

Treatment advances for Duchenne muscular dystrophy.

Zygmunt A, Tian C

Unexpected sudden cardiac arrest due to ventricular fibrillation in a 7-year-old boy with Duchenne muscular dystrophy.

Mhanna C, Nees S, Tsuda T

Correction: Incremental Disease Burden (Healthcare Costs and Resources) of Duchenne Muscular Dystrophy in the US: A Matched Cohort Analysis.

Scavina M, Posner N, Dorling P et al.

The burden of bone disease in Duchenne muscular dystrophy: age-specific prevalence of osteoporosis and low bone density.

Nasomyont N, Tian C, Hornung L et al.

Cognitive deficits linked to intrinsic timescales and gray matter volume abnormalities in children with Duchenne muscular dystrophy.

Niu X, Hu Q, Zhang X et al.

Anti-Inflammatory and Synaptic Protective Effects of TNF-α Inactivation in the MDX Mouse Model.

Bonani AO, Matheus V, Tomiyama ALMR et al.

Loss of dystrophin reduces CB1 receptor expression and endocannabinoid-dependent synaptic plasticity in the cerebellar cortex.

Averyt E, Mitra S, Pugh JR

Measuring health-related quality of life in facioscapulohumeral muscular dystrophy: a COSMIN systematic review and conceptual framework.

Carlton J, Powell P, Street J et al.

Living with Duchenne muscular dystrophy as an adult: motor function, ventilatory assistance and health-related quality of life.

Wahlgren L, Nordström S, Kroksmark AK et al.