Medicine

Evolving ASO therapies from advancement to implementation

.Contending enthusiasms.R.S., M.S., H.G. and A.A.R. are actually organizers of the 1M1M project. H.G. and also A.A.R. are board of directors members and also R.S., M.S. as well as A.A.R. are participants of the clinical advisory committee of N1C. A.A.R. discloses employment through LUMC, which possesses patents on exon-skipping technology, a number of which has actually been actually accredited to BioMarin and also consequently sublicensed to Sarepta. As co-inventor of a number of these patents, A.A.R. was allowed to a share of nobilities. A.A.R. better discloses functioning as impromptu professional for PTC Therapeutics, Sarepta Rehabs, Regenxbio, Dyne Therapies, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. In the past 5 years, A.A.R. also conducted impromptu speaking to for Alpha Anomeric. A.A.R. likewise mentions membership of the scientific advisory boards of Eisai, Hybridize Therapeutics, Silence Rehabs, Sarepta Rehabs, Sapreme as well as Mitorx. Over the last 5 years, A.A.R. was actually additionally a clinical advisory board member for ProQR. Reimbursement for A.A.R. u00e2 s consulting as well as advising tasks is paid for to LUMC. Over the last 5 years, LUMC likewise acquired speaker gratuity coming from PTC Therapeutics, Alnylam Netherlands, Italfarmaco as well as Pfizer as well as moneying for contract investigation coming from Sapreme, Eisai, Galapagos, Synaffix and also Alpha Anomeric. Task backing is obtained coming from Sarepta Therapies and Entrada by means of unrestricted gives. H.G. has nothing at all to reveal in connection with the topics covered within this composition. In the past 5 years, he has likewise gotten working as a consultant honoraria coming from UCB. M.S. received consultancy gratuity coming from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and Solaxa previously 5 years, all irrelevant to today document. R.S. has nothing to make known in relation to the subject matters covered within this document. She has actually obtained sound speaker and/or working as a consultant honoraria or even sponsoring additions coming from Abbvie, Bial, STADA as well as Everpharma before 5 years.