Medicine

Progressing ASO treatments coming from development to application

.Contending passions.R.S., M.S., H.G. and also A.A.R. are coordinators of the 1M1M campaign. H.G. and A.A.R. are actually panel of directors participants and also R.S., M.S. as well as A.A.R. are actually members of the medical advising committee of N1C. A.A.R. divulges work through LUMC, which has patents on exon-skipping technology, several of which has been actually licensed to BioMarin as well as subsequently sublicensed to Sarepta. As co-inventor of several of these patents, A.A.R. was actually entitled to a share of royalties. A.A.R. even more makes known serving as ad hoc specialist for PTC Therapeutics, Sarepta Rehabs, Regenxbio, Dyne Therapies, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and Astra Zeneca. Before 5 years, A.A.R. also carried out impromptu seeking advice from for Alpha Anomeric. A.A.R. also states registration of the clinical boards of advisers of Eisai, Hybridize Therapies, Silence Rehabs, Sarepta Rehabs, Sapreme and Mitorx. Previously 5 years, A.A.R. was actually likewise a clinical advisory board member for ProQR. Wage for A.A.R. u00e2 s consulting and also encouraging tasks is paid for to LUMC. Over the last 5 years, LUMC likewise obtained speaker gratuity coming from PTC Rehabs, Alnylam Netherlands, Italfarmaco and also Pfizer and moneying for deal research coming from Sapreme, Eisai, Galapagos, Synaffix and Alpha Anomeric. Project funding is acquired from Sarepta Rehabs and Entrada through unlimited grants. H.G. has nothing at all to divulge in regard to the subjects covered within this manuscript. Before 5 years, he has also received consultancy honoraria from UCB. M.S. got working as a consultant honoraria coming from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and also Solaxa over the last 5 years, all unassociated to today document. R.S. possesses nothing to disclose in connection with the topics dealt with in this manuscript. She has actually received audio speaker and/or working as a consultant gratuity or even sponsoring additions coming from Abbvie, Bial, STADA and Everpharma in the past 5 years.