Medicine

Accelerating ASO treatments from development to execution

.Contending interests.R.S., M.S., H.G. and also A.A.R. are actually coordinators of the 1M1M campaign. H.G. and also A.A.R. are board of directors participants and R.S., M.S. as well as A.A.R. are actually members of the clinical advisory board of N1C. A.A.R. discloses work by LUMC, which possesses patents on exon-skipping modern technology, a few of which has been certified to BioMarin and consequently sublicensed to Sarepta. As co-inventor of some of these patents, A.A.R. was qualified to an allotment of nobilities. A.A.R. additionally discloses serving as professional for PTC Therapies, Sarepta Therapies, Regenxbio, Dyne Therapies, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. Over the last 5 years, A.A.R. also executed ad hoc consulting for Alpha Anomeric. A.A.R. also discloses registration of the scientific boards of advisers of Eisai, Hybridize Rehabs, Muteness Therapies, Sarepta Therapeutics, Sapreme and also Mitorx. In the past 5 years, A.A.R. was likewise a scientific board of advisers member for ProQR. Commission for A.A.R. u00e2 s consulting as well as urging tasks is actually paid for to LUMC. Previously 5 years, LUMC additionally got sound speaker honoraria coming from PTC Therapies, Alnylam Netherlands, Italfarmaco and Pfizer as well as funding for arrangement analysis from Sapreme, Eisai, Galapagos, Synaffix and also Alpha Anomeric. Project financing is actually obtained from Sarepta Rehabs and also Entrada using unconstrained grants. H.G. possesses absolutely nothing to make known in relation to the subject matters dealt with within this composition. In the past 5 years, he has also obtained consultancy honoraria coming from UCB. M.S. obtained working as a consultant gratuity from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly as well as Solaxa over the last 5 years, all unconnected to today composition. R.S. possesses nothing at all to reveal in relation to the subject matters dealt with in this particular manuscript. She has acquired sound speaker and/or working as a consultant gratuity or financing additions from Abbvie, Bial, STADA and Everpharma previously 5 years.