Disclosures

Disclosures of Annemieke Aartsma-Rus PhD, Professor of Translational Genetics

Annemieke Aartsma-Rus discloses being employed by LUMC which has patents on exon skipping technology, some of which has been licensed to BioMarin and subsequently sublicensed to Sarepta. As co-inventor of some of these patents, AAR is entitled to a share of royalties.

Current scientific advisory board memberships: Sarepta Therapeutics, Silence, Eisai, Hybridize therapeutics

Past scientific advisory board memberships (<5 years ago): ProQR, Philae Pharmaceuticals

Current ad hoc consulting: PTC Therapeutics, Sarepta Therapeutics, Alpha Anomeric, BioMarin Pharmaceuticals Inc, Eisai, Entrada, Takeda, Splicesense, Galapagos, Audentes,

Past ad hoc consulting (<5 years ago): CRISPR Therapeutics, Summit PLC, Astra Zeneca, Santhera, Bridge Bio, Global Guidepoint, GLG consulting, Grunenthal, Wave Life Sciences, Bioclinica

Remuneration for advisory board and consulting activities and speaker honoraria (form PTC Therapeutics) all go to LUMC. No personal financial compensation is received by Professor Aartsma-Rus for these activities.

LUMC receives funding for contract research from Italpharmaco and Alpha Anomeric conducted by the group of Prof Aartsma-Rus. Project funding is received from Sarepta Therapeutics via unrestricted grants.