M. Weiler

Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study

B. Schlotter-Weigel, M. Blankenburg, P. Huppke, 2022, Orphanet Journal of Rare Diseases.

Improvements in Walking Distance during Nusinersen Treatment – A Prospective 3-year SMArtCARE Registry Study

S. Petri, A. Blaschek, B. Schrank, 2022, Journal of neuromuscular diseases.

Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

A. Ludolph, T. Hagenacker, P. Weydt, 2022, Orphanet Journal of Rare Diseases.

Biomarkers in 5q-associated spinal muscular atrophy—a narrative review

T. Hagenacker, C. Wurster, R. Günther, 2023, Journal of Neurology.

Evaluation of risdiplam efficacy in 5q spinal muscular atrophy: A systematic comparison of electrophysiologic with clinical outcome measures

Georges Sam, T. Kessler, W. Wick, 2023, European journal of neurology.

Prognostic Value of Standard Heart Failure Medication in Patients with Cardiac Transthyretin Amyloidosis

U. Hegenbart, A. Kristen, M. Weiler, 2024, Journal of clinical medicine.

Correction to: Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

A. Ludolph, T. Hagenacker, P. Weydt, 2022, Orphanet Journal of Rare Diseases.