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2017 1
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204 results

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Page 1
Risdiplam in Type 1 Spinal Muscular Atrophy.
Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Baranello G, et al. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. N Engl J Med. 2021. PMID: 33626251 Clinical Trial.
Crovalimab treatment in patients with paroxysmal nocturnal haemoglobinuria: Long-term results from the phase I/II COMPOSER trial.
Röth A, Ichikawa S, Ito Y, Kim JS, Nagy Z, Obara N, Panse J, Schrezenmeier H, Sica S, Soret J, Usuki K, Yoon SS, Balachandran N, Buri M, Lundberg P, Patel H, Shinomiya K, Sostelly A, Nishimura JI. Röth A, et al. Eur J Haematol. 2023 Aug;111(2):300-310. doi: 10.1111/ejh.14011. Epub 2023 Jun 15. Eur J Haematol. 2023. PMID: 37321625 Clinical Trial.
Efficacy, durability, and safety of faricimab up to every 16 weeks in patients with neovascular age-related macular degeneration: 1-year results from the Japan subgroup of the phase 3 TENAYA trial.
Mori R, Honda S, Gomi F, Tsujikawa A, Koizumi H, Ochi H, Ohsawa S, Okada AA; TENAYA and LUCERNE Investigators. Mori R, et al. Jpn J Ophthalmol. 2023 May;67(3):301-310. doi: 10.1007/s10384-023-00985-w. Epub 2023 Apr 11. Jpn J Ophthalmol. 2023. PMID: 37039948 Free PMC article. Clinical Trial.
A randomized, double-blind phase 1b study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of the NLRP3 inhibitor selnoflast in patients with moderate to severe active ulcerative colitis.
Klughammer B, Piali L, Nica A, Nagel S, Bailey L, Jochum C, Ignatenko S, Bläuer A, Danilin S, Gulati P, Hayward J, Scepanovic P, Zhang JD, Bhosale S, Chong CF, Christ A. Klughammer B, et al. Clin Transl Med. 2023 Nov;13(11):e1471. doi: 10.1002/ctm2.1471. Clin Transl Med. 2023. PMID: 37962000 Free PMC article. Clinical Trial.
204 results