Updates are available Correction dated 2022-10-25

Article History

Received: 7 February 2022

Accepted: 30 June 2022

First Online: 19 July 2022

Change Date: 25 October 2022

Change Type: Correction

Change Details: A Correction to this paper has been published:

Change Details: https://doi.org/10.1186/s13023-022-02548-7

Declarations

:

: The initial notification for this CUP was submitted on January 30, 2020, to the Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM), followed by a written confirmation of a valid notification of this CUP issued by BfArM on March 12, 2020. All patients and/or legally authorized representatives provided written informed consent to participate in the CUP.

: Not applicable. All patients and/or legally authorized representatives have signed a consent form in which they agreed to pseudonymized data being collected, analyzed and used in scientific publications.

: All authors received institutional support for their contribution to this CUP as well as medical writing support for publication of the data, both funded by Roche Pharma AG, Grenzach-Wyhlen, Germany. AH has received honoraria from Roche for participation at advisory boards and lectures. RG has received research grants from Biogen, personal fees and non-financial support from Biogen for lectures and for serving on advisory boards as well as personal fees from Hoffmann-La Roche for serving on advisory boards outside of the submitted work. AL has served on advisory boards for Roche Pharma AG. OS has declared no further competing interests. RT has received personal fees from Novartis, Sanofi, PTC, Biogen and Desitin for lectures and for serving on advisory boards outside of the submitted work. PW has received consulting fees from Roche, Biogen and ITF Pharma. MW advises for Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, Pfizer, Hoffmann-La Roche and Swedish Orphan Biovitrum, received lecture honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals and Biogen, and received financial support for conference attendance from Biogen and Pfizer, all outside of the present work. KN and MSS are employees of Roche Pharma AG. TH has received research grants and personal fees for lectures from Biogen, Novartis Gene Therapies, Sanofi-Genzyme and Hoffmann-La Roche and for serving on advisory boards from Biogen, Sanofi-Genzyme, Hoffmann-La Roche, Alexion, Hormosan, Alnylam, Argenx and PTC outside the submitted work.