Medicine

Evolving ASO therapies coming from development to execution

.Competing rate of interests.R.S., M.S., H.G. and also A.A.R. are actually planners of the 1M1M initiative. H.G. and also A.A.R. are panel of supervisors members as well as R.S., M.S. and also A.A.R. are participants of the medical advising committee of N1C. A.A.R. divulges work by LUMC, which possesses patents on exon-skipping modern technology, a number of which has been actually licensed to BioMarin as well as ultimately sublicensed to Sarepta. As co-inventor of a few of these patents, A.A.R. was actually qualified to a reveal of royalties. A.A.R. further reveals acting as expert for PTC Rehabs, 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 carried out speaking to for Alpha Anomeric. A.A.R. additionally discloses subscription of the clinical advisory boards of Eisai, Hybridize Therapeutics, Silence Therapies, Sarepta Rehabs, Sapreme and Mitorx. Previously 5 years, A.A.R. was likewise a clinical board of advisers participant for ProQR. Pay for A.A.R. u00e2 s consulting and advising activities is paid to LUMC. Over the last 5 years, LUMC likewise obtained sound speaker gratuity coming from PTC Therapies, Alnylam Netherlands, Italfarmaco and also Pfizer and also cashing for contract research study coming from Sapreme, Eisai, Galapagos, Synaffix and Alpha Anomeric. Project funding is actually received coming from Sarepta Therapeutics as well as Entrada through unrestricted grants. H.G. possesses nothing at all to divulge in regard to the subject matters dealt with in this particular composition. In the past 5 years, he has additionally obtained working as a consultant honoraria from UCB. M.S. acquired working as a consultant gratuity from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and Solaxa over the last 5 years, all unrelated to the here and now composition. R.S. has nothing to make known in regard to the subject matters covered within this manuscript. She has received sound speaker and/or working as a consultant gratuity or even funding additions coming from Abbvie, Bial, STADA as well as Everpharma over the last 5 years.

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