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Page 1
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
McMillan HJ, Baranello G, Farrar MA, Zaidman CM, Moreno T, De Waele L, Jong YJ, Laugel V, Quijano-Roy S, Mercuri E, Chien YH, Straub V, Darras BT, Seibert J, Bernardo Escudero R, Alecu I, Freischläger F, Muntoni F; SMART Study Group. McMillan HJ, et al. Neurology. 2025 Jan 28;104(2):e210268. doi: 10.1212/WNL.0000000000210268. Epub 2024 Dec 30. Neurology. 2025. PMID: 39804575 Free PMC article. Clinical Trial.
Therapeutic phlebotomy is safe in children with sickle cell anaemia and can be effective treatment for transfusional iron overload.
Aygun B, Mortier NA, Kesler K, Lockhart A, Schultz WH, Cohen AR, Alvarez O, Rogers ZR, Kwiatkowski JL, Miller ST, Sylvestre P, Iyer R, Lane PA, Ware RE; Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) Trial Investigators. Aygun B, et al. Br J Haematol. 2015 Apr;169(2):262-6. doi: 10.1111/bjh.13280. Epub 2015 Jan 22. Br J Haematol. 2015. PMID: 25612463 Free PMC article. Clinical Trial.
Pain and other non-neurological adverse events in children with sickle cell anemia and previous stroke who received hydroxyurea and phlebotomy or chronic transfusions and chelation: results from the SWiTCH clinical trial.
Alvarez O, Yovetich NA, Scott JP, Owen W, Miller ST, Schultz W, Lockhart A, Aygun B, Flanagan J, Bonner M, Mueller BU, Ware RE; Investigators of the Stroke With Transfusions Changing to Hydroxyurea Clinical Trial (SWiTCH). Alvarez O, et al. Am J Hematol. 2013 Nov;88(11):932-8. doi: 10.1002/ajh.23547. Epub 2013 Aug 30. Am J Hematol. 2013. PMID: 23861242 Free PMC article. Clinical Trial.
19 results