Janbernd Kirschner

Newbornscreening SMA - from Pilot Project to Nationwide Screening in Germany

Abstract: Now that targeted therapies for spinal muscular atrophy are available, attempts are being made worldwide to include screening for spinal mus...

Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen Within the Expanded Access Program in Germany

Abstract: Background: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by muscle weakness and muscle atrophy. Nusinersen acts a...

Tapering Canakinumab Monotherapy in Patients with Systemic Juvenile Idiopathic Arthritis in Clinical Remission: Results from a Phase IIIb/IV Open-label, Randomized Study

Abstract: Objective To evaluate the efficacy and safety of 2 canakinumab monotherapy tapering regimens in order to maintain complete clinical remissio...