Therapeutic strategies in congenital myasthenic syndromes

per Schara, Ulrike, Lochmüller, Hanns
Publicat a Neurotherapeutics (2008)

New treatments for neuromuscular disease: Optimism and obstacles

per Lochmüller, Hanns, Griggs, Robert C.
Publicat a Neurotherapeutics (2008)

Congenital Myasthenic Syndromes with Predominant Limb Girdle Weakness

per Evangelista, Teresinha, Hanna, Mike, Lochmüller, Hanns
Publicat a J Neuromuscul Dis (2015)

Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy

per Landfeldt, Erik, Lochmüller, Hanns, Lindgren, Peter
Publicat a Orphanet J Rare Dis (2019)

Transition from childhood to adulthood in Duchenne muscular dystrophy (DMD)

per Rodger, Sunil, Steffensen, Birgit F, Lochmüller, Hanns
Publicat 2012

Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders

per Lochmüller, Hanns, Ramirez, Antonio Nino, Kakkis, Emil
Publicat a Orphanet J Rare Dis (2021)

Targeted therapies for congenital myasthenic syndromes: systematic review and steps towards a treatabolome

per Thompson, Rachel, Bonne, Gisèle, Missier, Paolo, Lochmüller, Hanns
Publicat a Emerg Top Life Sci (2019)

215th ENMC International Workshop VCP-related multi-system proteinopathy (IBMPFD) 13–15 November 2015, Heemskerk, The Netherlands

per Evangelista, Teresinha, Weihl, Conrad C., Kimonis, Virginia, Lochmüller, Hanns
Publicat a Neuromuscul Disord (2016)

Linker molecules between laminins and dystroglycan ameliorate laminin-α2–deficient muscular dystrophy at all disease stages

per Meinen, Sarina, Barzaghi, Patrizia, Lin, Shuo, Lochmüller, Hanns, Ruegg, Markus A.
Publicat 2007

How reference networks develop, implement, and monitor guidelines

per Kirschner, Jan, Rodger, Sunil, Vry, Julia, Gramsch, Kathrin, Lochmüller, Hanns, Bushby, Kate
Publicat 2012

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

per Greer, Kane L, Lochmüller, Hanns, Flanigan, Kevin, Fletcher, Susan, Wilton, Steve D
Publicat 2014

Human Skeletal Muscle–derived CD133(+) Cells Form Functional Satellite Cells After Intramuscular Transplantation in Immunodeficient Host Mice

per Meng, Jinhong, Chun, Soyon, Asfahani, Rowan, Lochmüller, Hanns, Muntoni, Francesco, Morgan, Jennifer
Publicat 2014

Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis

per Landfeldt, Erik, Alfredsson, Lars, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine, Lindgren, Peter
Publicat a Pharmacoeconomics (2016)

Congenital Myasthenic Syndromes or Inherited Disorders of Neuromuscular Transmission: Recent Discoveries and Open Questions

per Nicole, Sophie, Azuma, Yoshiteru, Bauché, Stéphanie, Eymard, Bruno, Lochmüller, Hanns, Slater, Clarke
Publicat a J Neuromuscul Dis (2017)

GNE myopathy: from clinics and genetics to pathology and research strategies

per Pogoryelova, Oksana, González Coraspe, José Andrés, Nikolenko, Nikoletta, Lochmüller, Hanns, Roos, Andreas
Publicat a Orphanet J Rare Dis (2018)

An improved method for culturing myotubes on laminins for the robust clustering of postsynaptic machinery

per Pęziński, Marcin, Daszczuk, Patrycja, Pradhan, Bhola Shankar, Lochmüller, Hanns, Prószyński, Tomasz J.
Publicat a Sci Rep (2020)

Costs of Illness of Spinal Muscular Atrophy: A Systematic Review

per Landfeldt, Erik, Pechmann, Astrid, McMillan, Hugh J., Lochmüller, Hanns, Sejersen, Thomas
Publicat a Appl Health Econ Health Policy (2021)

Inclusion Body Myositis: Laser Microdissection Reveals Differential Up-Regulation of IFN-γ Signaling Cascade in Attacked versus Nonattacked Myofibers

per Ivanidze, Jana, Hoffmann, Reinhard, Lochmüller, Hanns, Engel, Andrew G., Hohlfeld, Reinhard, Dornmair, Klaus
Publicat 2011

Whole-exome sequencing identifies mutations in MYMK in a mild form of Carey-Fineman-Ziter syndrome

per Alrohaif, Hadil, Töpf, Ana, Evangelista, Teresinha, Lek, Monkol, McArthur, Daniel, Lochmüller, Hanns
Publicat a Neurol Genet (2018)

The beta-adrenergic agonist salbutamol modulates neuromuscular junction formation in zebrafish models of human myasthenic syndromes

per McMacken, Grace, Cox, Dan, Roos, Andreas, Müller, Juliane, Whittaker, Roger, Lochmüller, Hanns
Publicat a Hum Mol Genet (2018)