Medicine

Progressing ASO treatments coming from progression to implementation

.Contending passions.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 supervisors members and R.S., M.S. and A.A.R. are actually members of the medical advisory committee of N1C. A.A.R. makes known work through LUMC, which possesses licenses on exon-skipping technology, a few of which has actually been certified to BioMarin as well as ultimately sublicensed to Sarepta. As co-inventor of a few of these patents, A.A.R. was actually entitled to a portion of royalties. A.A.R. further reveals functioning as ad hoc professional for PTC Rehabs, Sarepta Therapeutics, Regenxbio, Dyne Rehabs, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. Before 5 years, A.A.R. additionally conducted seeking advice from for Alpha Anomeric. A.A.R. also states subscription of the scientific boards of advisers of Eisai, Hybridize Rehabs, Muteness Therapies, Sarepta Therapeutics, Sapreme as well as Mitorx. Before 5 years, A.A.R. was likewise a medical advisory board participant for ProQR. Reimbursement for A.A.R. u00e2 s consulting and also suggesting activities is paid out to LUMC. Before 5 years, LUMC likewise received sound speaker gratuity coming from PTC Therapeutics, Alnylam Netherlands, Italfarmaco as well as Pfizer as well as cashing for deal analysis coming from Sapreme, Eisai, Galapagos, Synaffix as well as Alpha Anomeric. Task backing is gotten from Sarepta Therapeutics and also Entrada through unrestricted gives. H.G. possesses nothing at all to reveal in regard to the subject matters dealt with in this particular document. Over the last 5 years, he has likewise obtained working as a consultant gratuity coming from UCB. M.S. got consultancy honoraria coming from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly as well as Solaxa in the past 5 years, all unconnected to the present manuscript. R.S. possesses nothing to disclose in relation to the subject matters dealt with within this document. She has received sound speaker and/or consultancy honoraria or funding contributions coming from Abbvie, Bial, STADA as well as Everpharma before 5 years.