Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.

"CONFLICT OF INTEREST Craig M. McDonald has acted as a consultant on clinical trials of DMD for Astellas, Capricor, Catabasis, Edgewise Therapeutics, Epirium Bio (formerly Cardero Therapeutics), FibroGen, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. He has received research support for clinical trials from Capricor, Catabasis, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. Lee‐Jen Wei has acted as a consultant for Johnson and Johnson, Merck and co., Pfizer, PTC Therapeutics and Sarepta Therapeutics. Kevin M. Flanigan has acted as a consultant for Apic Bio, Audentes Therapeutics, Dynacure, Italfarmaco, Marathon Pharmaceuticals, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics, Tivorsan Pharmaceuticals, and 4D Molecular Therapeutics; has been a clinical trial investigator for Abeona Therapeutics, Akashi Therapeutics BioMarin and Sarepta Therapeutics; has received grants from Beauhawks Foundation and CureDuchenne; and has received royalty payment from Audentes Therapeutics. Gary Elfring and Panayiota Trifillis are employees of PTC Therapeutics. Francesco Muntoni has received consulting fees from AveXis, Biogen, Dyne Therapeutics, Capricor, Catabasis, Novartis, Pfizer, PTC Therapeutics, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics and Wave Therapeutics, and is supported by the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. Medical writing and editorial support were funded by PTC Therapeutics Ltd."

"Funding information PTC Therapeutics, Inc"