Johannes Friese
Bonn, NW, Germany
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 12 publications
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.
Journal: Journal of neuromuscular diseases
Published: August 27, 2026
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence.
Journal: Journal of neuromuscular diseases
Published: November 24, 2025
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec.
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: November 06, 2024
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.
Journal: The Lancet regional health. Europe
Published: June 28, 2024
Phenylbutyrate Treatment in a Boy With MCT8 Deficiency: Improvement of Thyroid Function Tests and Possible Hepatotoxicity.
Journal: The Journal of clinical endocrinology and metabolism
Published: February 09, 2024
Troponin T is elevated in a relevant proportion of patients with 5q-associated spinal muscular atrophy.
Journal: Scientific reports
Published: November 07, 2023
5qSMA: standardised retrospective natural history assessment in 268 patients with four copies of SMN2.
Journal: Journal of neurology
Published: October 30, 2023
Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.
Journal: Journal of neuromuscular diseases
Published: December 24, 2022
Newbornscreening SMA - From Pilot Project to Nationwide Screening in Germany.
Journal: Journal of neuromuscular diseases
Published: December 04, 2022
Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study.
Journal: Orphanet journal of rare diseases
Published: June 23, 2022
Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec -A Single Centre Experience.
Journal: Journal of neuromuscular diseases
Published: January 11, 2021
Showing 1-12 of 12
Last Updated: 09/26/2026