Document is current

Article History

Accepted: 19 September 2024

First Online: 4 November 2024

Competing interests

: A.A.-R. is an unpaid board member of the Dutch Centre for RNA Therapeutics and an unpaid board member of the <i>N</i> = 1 Collaborative (N1C). A.B.-H. is an uncompensated volunteer for the n-Lorem Foundation’s Access to Treatment committee. A.J. has received speaking fees from Vertex Biopharmaceuticals. D.D. is an employee of Creyon Bio. H.G. is a Member of the Board of Directors of the N1C. D.O.’C. is involved in the Rare Therapies Launch Pad (RTLP) as representative for the ABPI, a non-financial interaction. J.Da. is co-founder of Nucleome Therapeutics and provides consultancy to the company; and has intellectual property licensed to BEAM therapeutics, receives revenue from this licence and holds personal shares. M.S. has received consultancy honoraria from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and Solaxa, all unrelated to the present manuscript; and is an unpaid board member of the N1C. R.H. is CEO and founder of Cure Rare Disease; CEO and founder of Myofinity Biosciences; and Consultant for Neuromuscular Disease Foundation. T.Y. has received research support for an <i>N</i> = 1 trial in ataxia telangiectasia from EveryONE Medicines, and is named as an inventor on three patents pertaining to antisense oligonucleotide (ASO) treatments for transposon-associated diseases (WO2019055460A1), ataxia telangiectasia (US20230174979A1) and progranulin (US11359199B2); has received scientific consulting fees as pertains to ASO therapies from GeneTx, RegUp and Servier Pharmaceuticals; serves as an unpaid Board Member for the N1C, the Oligonucleotide Therapeutics Society and the Society for RNA Therapeutics; and serves as an unpaid scientific adviser to several rare disease foundations. A.H.-J., E.-A.T, G.B., S.M., O.I., E.F.A., A.M.G.P., J.D., L.L. and P.J.B. declare no competing interests.