Medicine

Progressing ASO treatments from progression to implementation

.Contending rate of interests.R.S., M.S., H.G. and also A.A.R. are actually coordinators of the 1M1M initiative. H.G. and also A.A.R. are actually board of supervisors participants and also R.S., M.S. as well as A.A.R. are actually members of the clinical consultatory committee of N1C. A.A.R. discloses employment through LUMC, which has licenses on exon-skipping technology, several of which has been actually certified to BioMarin and consequently sublicensed to Sarepta. As co-inventor of several of these licenses, A.A.R. was qualified to a reveal of nobilities. A.A.R. better reveals working as ad hoc consultant for PTC Therapies, Sarepta Rehabs, Regenxbio, Dyne Therapeutics, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. In the past 5 years, A.A.R. additionally performed speaking with for Alpha Anomeric. A.A.R. likewise states membership of the clinical boards of advisers of Eisai, Hybridize Therapeutics, Silence Therapeutics, Sarepta Therapies, Sapreme as well as Mitorx. In the past 5 years, A.A.R. was likewise a scientific board of advisers participant for ProQR. Compensation for A.A.R. u00e2 s consulting and also urging activities is spent to LUMC. Previously 5 years, LUMC additionally acquired sound speaker honoraria from PTC Therapeutics, Alnylam Netherlands, Italfarmaco and Pfizer and also funding for agreement analysis coming from Sapreme, Eisai, Galapagos, Synaffix and also Alpha Anomeric. Job backing is received coming from Sarepta Therapeutics and Entrada by means of unconstrained gives. H.G. has nothing to disclose relative to the subjects dealt with in this composition. Before 5 years, he has additionally acquired consultancy gratuity coming from UCB. M.S. obtained working as a consultant honoraria from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly as well as Solaxa before 5 years, all unassociated to the present composition. R.S. possesses nothing to disclose in connection with the subject matters dealt with within this manuscript. She has actually received audio speaker and/or working as a consultant honoraria or even funding additions coming from Abbvie, Bial, STADA and also Everpharma over the last 5 years.