Medicine

Progressing ASO treatments from development to application

.Competing passions.R.S., M.S., H.G. and also A.A.R. are coordinators of the 1M1M initiative. H.G. and also A.A.R. are board of supervisors members and R.S., M.S. and also A.A.R. are actually participants of the clinical advising committee of N1C. A.A.R. discloses work through LUMC, which has licenses on exon-skipping technology, some of which has actually been licensed 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. even further divulges acting as impromptu expert for PTC Rehabs, Sarepta Therapies, Regenxbio, Dyne Therapeutics, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and Astra Zeneca. Over the last 5 years, A.A.R. additionally performed ad hoc consulting for Alpha Anomeric. A.A.R. also reports membership of the clinical boards of advisers of Eisai, Hybridize Therapies, Muteness Rehabs, Sarepta Therapies, Sapreme and Mitorx. Over the last 5 years, A.A.R. was likewise a clinical advisory board participant for ProQR. Commission for A.A.R. u00e2 s consulting and recommending tasks is paid to LUMC. In the past 5 years, LUMC also obtained sound speaker gratuity from PTC Therapies, Alnylam Netherlands, Italfarmaco and also Pfizer as well as cashing for deal investigation from Sapreme, Eisai, Galapagos, Synaffix and also Alpha Anomeric. Task financing is received coming from Sarepta Therapeutics as well as Entrada through unlimited gives. H.G. possesses nothing to reveal relative to the subject matters covered within this manuscript. Previously 5 years, he has actually additionally obtained working as a consultant gratuity coming from UCB. M.S. received working as a consultant gratuity from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and also Solaxa before 5 years, all unassociated to the present manuscript. R.S. has nothing at all to reveal relative to the topics covered in this composition. She has actually gotten sound speaker and/or working as a consultant gratuity or even funding additions from Abbvie, Bial, STADA and Everpharma previously 5 years.

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