Accelerating ASO treatments from development to application

.Completing interests.R.S., M.S., H.G. and A.A.R. are actually coordinators of the 1M1M initiative.

H.G. as well as A.A.R. are board of supervisors participants and R.S., M.S.

as well as A.A.R. are actually participants of the medical advising board of N1C. A.A.R.

makes known work by LUMC, which possesses licenses on exon-skipping modern technology, some of which has been certified to BioMarin and ultimately sublicensed to Sarepta. As co-inventor of several of these patents, A.A.R. was actually allowed to an allotment of royalties.

A.A.R. additionally makes known acting as specialist for PTC Therapeutics, Sarepta Rehabs, Regenxbio, Dyne Rehabs, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. In the past 5 years, A.A.R.

also conducted ad hoc speaking with for Alpha Anomeric. A.A.R. likewise mentions membership of the scientific boards of advisers of Eisai, Hybridize Therapies, Silence Therapeutics, Sarepta Rehabs, Sapreme as well as Mitorx.

Before 5 years, A.A.R. was actually likewise a medical board of advisers member for ProQR. Compensation for A.A.R.

u00e2 s consulting as well as advising tasks is paid for to LUMC. Before 5 years, LUMC additionally received sound speaker honoraria from PTC Therapies, Alnylam Netherlands, Italfarmaco and also Pfizer and also funding for deal investigation from Sapreme, Eisai, Galapagos, Synaffix and also Alpha Anomeric. Venture financing is actually received coming from Sarepta Therapeutics and Entrada via unrestricted grants.

H.G. possesses nothing to disclose in connection with the subjects covered within this manuscript. Over the last 5 years, he has actually additionally gotten working as a consultant honoraria coming from UCB.

M.S. acquired consultancy honoraria coming from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and Solaxa over the last 5 years, all unassociated to today document. R.S.

has absolutely nothing to divulge in regard to the subject matters dealt with in this manuscript. She has obtained speaker and/or working as a consultant gratuity or financing additions from Abbvie, Bial, STADA as well as Everpharma before 5 years.