Medicine

Evolving ASO treatments coming from development to application

.Contending passions.R.S., M.S., H.G. and also A.A.R. are actually organizers of the 1M1M initiative. H.G. as well as A.A.R. are actually board of supervisors members and R.S., M.S. and also A.A.R. are members of the clinical advising committee of N1C. A.A.R. discloses job by LUMC, which has licenses on exon-skipping technology, some of which has actually been actually accredited to BioMarin and consequently sublicensed to Sarepta. As co-inventor of some of these licenses, A.A.R. was allowed to a share of royalties. A.A.R. additionally discloses serving as ad hoc consultant for PTC Therapeutics, Sarepta Rehabs, Regenxbio, Dyne Rehabs, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco as well as Astra Zeneca. In the past 5 years, A.A.R. likewise carried out consulting for Alpha Anomeric. A.A.R. additionally states subscription of the scientific advisory boards of Eisai, Hybridize Therapies, Muteness Therapeutics, Sarepta Rehabs, Sapreme as well as Mitorx. In the past 5 years, A.A.R. was likewise a clinical advisory board member for ProQR. Reimbursement for A.A.R. u00e2 s consulting and also advising activities is actually paid out to LUMC. Over the last 5 years, LUMC additionally obtained sound speaker honoraria coming from PTC Therapeutics, Alnylam Netherlands, Italfarmaco and also Pfizer and funding for contract research from Sapreme, Eisai, Galapagos, Synaffix as well as Alpha Anomeric. Project funding is received from Sarepta Therapies as well as Entrada through unconstrained grants. H.G. has nothing at all to disclose in connection with the subjects covered in this document. Over the last 5 years, he has also gotten consultancy honoraria from UCB. M.S. acquired working as a consultant gratuity coming from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and also Solaxa over the last 5 years, all unrelated to the here and now composition. R.S. possesses nothing to reveal in regard to the topics dealt with within this manuscript. She has actually acquired sound speaker and/or consultancy honoraria or even financing additions coming from Abbvie, Bial, STADA and also Everpharma before 5 years.

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