Wolfgang Müller‐Felber

LMU Klinikum — Germany

Medicine · Health Sciences

46h-index6.4kcitations269works15.42yr avg
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Research Topics

Neurogenetic and Muscular Disorders Research(65), Muscle Physiology and Disorders(47), Hereditary Neurological Disorders(23), Genetic Neurodegenerative Diseases(22), Glycogen Storage Diseases and Myoclonus(22)

Publications264 total

Neuromuskuläre Erkrankungen bei Kindern und Jugendlichen
2025
Phenotypic intrafamilial variability of 5q-associated spinal muscular atrophy: A systematic multicentre sibling study
Journal of Neuromuscular Diseases·2025· 1 citedOpen Access
03OCognitive development in children with 5q-SMA identified by neonatal screening - 4 years follow-up
Neuromuscular Disorders·2025
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study
Therapeutic Advances in Rare Disease·2025Open Access
Pädiatrische Neurologie - Neuropädiatrie
2025
Familienbelastung von Familien mit Kindern mit neuromuskulärer Erkrankung und Langzeitbeatmung
Zeitschrift für Geburtshilfe und Neonatologie·2025
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial
Obstetrical & Gynecological Survey·2024· 2 citedOpen Access
Identification of Biochemical Determinants for Diagnosis and Prediction of Severity in 5q Spinal Muscular Atrophy Using 1H-Nuclear Magnetic Resonance Metabolic Profiling in Patient-Derived Biofluids
International Journal of Molecular Sciences·2024· 3 citedOpen Access
Postnatal management of preterm infants with spinal muscular atrophy: experience from German newborn screening
Orphanet Journal of Rare Diseases·2024· 5 citedOpen Access
Long-Term Socioeconomic and Neurologic Outcome for Individuals with Childhood-Onset Multiple Sclerosis
Children·2024· 1 citedOpen Access
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy
JAMA Pediatrics·2024· 51 cited
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
The Lancet Neurology·2024· 100 cited
Biallelic variants in <i>SNUPN</i> cause a limb girdle muscular dystrophy with myofibrillar-like features
Brain·2024· 9 citedOpen Access
Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study
The Lancet Regional Health - Europe·2024· 55 citedOpen Access
Gentherapien bei monogenen Erkrankungen
Pädiatrie·2024
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
EBioMedicine·2023· 9 citedOpen Access
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
The Lancet Neurology·2023· 101 citedOpen Access
Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA
Neuropediatrics·2023
Impaired Neurodevelopment in Children with 5q-SMA - 2 Years After Newborn Screening
Journal of Neuromuscular Diseases·2023· 21 citedOpen Access
P206 Impaired neurodevelopment in children with 5q-SMA - 2 years after newborn screening
Neuromuscular Disorders·2023· 1 cited
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Frequent Co-authors

Hanns Lochmüller(17), D. Pongratz(10), Maggie C. Walter(10), Ulrike Schara(10), Benedikt Schoser(9), Astrid Blaschek(8), Marcus Deschauer(6), Katharina Vill(6), Angela Abicht(6), Rita Horváth(5), Peter Reilich(5), Heike Kölbel(5), Janbernd Kirschner(5), Rolf Stucka(5), Oliver Schwartz(4), Siegfried Burggraf(4), Wulf Röschinger(4), Jürgen Durner(4), Dieter Gläser(4), Brunhilde Wirth(4)