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Regulatory news: Nulibry (fosdenopterin) approved to reduce the risk of mortality in patients with molybdenum cofactor deficiency type A: FDA approval summary.
Farrell S, Karp J, Hager R, Wang Y, Adeniyi O, Wang J, Li L, Ma L, Peretz J, Summan M, Kong N, White M, Pacanowski M, Price D, Filie J, Donohue K, Joffe H. Farrell S, et al. Among authors: wang j, wang y. J Inherit Metab Dis. 2021 Sep;44(5):1085-1087. doi: 10.1002/jimd.12421. Epub 2021 Aug 8. J Inherit Metab Dis. 2021. PMID: 34337775 Review. No abstract available.
Dose-finding studies in drug development for rare genetic diseases.
Wang L, Wang J, Feng J, Doi M, Pepe S, Pacanowski M, Schuck RN. Wang L, et al. Among authors: wang j. Orphanet J Rare Dis. 2022 Apr 5;17(1):156. doi: 10.1186/s13023-022-02298-6. Orphanet J Rare Dis. 2022. PMID: 35382851 Free PMC article.
FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.
Suzuki M, Jeng LJB, Chefo S, Wang Y, Price D, Li X, Wang J, Li RJ, Ma L, Yang Y, Zhang X, Zheng N, Zhang K, Joseph DB, Shroff H, Doan J, Pacanowski M, Smpokou P, Donohue K, Joffe HV. Suzuki M, et al. Among authors: wang j, wang y. Genet Med. 2023 Feb;25(2):100335. doi: 10.1016/j.gim.2022.11.003. Epub 2022 Dec 12. Genet Med. 2023. PMID: 36507973 Free article.
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