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326 results

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Page 1
Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies.
Pane M, Berti B, Capasso A, Coratti G, Varone A, D'Amico A, Messina S, Masson R, Sansone VA, Donati MA, Agosto C, Bruno C, Ricci F, Pini A, Gagliardi D, Filosto M, Corti S, Leone D, Palermo C, Onesimo R, De Sanctis R, Ricci M, Bitetti I, Sframeli M, Dosi C, Albamonte E, Ticci C, Brolatti N, Bertini E, Finkel R, Mercuri E; ITASMAc group. Pane M, et al. Among authors: finkel r. EClinicalMedicine. 2023 May 5;59:101997. doi: 10.1016/j.eclinm.2023.101997. eCollection 2023 May. EClinicalMedicine. 2023. PMID: 37197706 Free PMC article.
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.
Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group. Victor RG, et al. Among authors: finkel r. Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29. Neurology. 2017. PMID: 28972192 Free PMC article. Clinical Trial.
Bartonella quintana Infection in Kidney Transplant Recipients from Donor Experiencing Homelessness, United States, 2022.
Beeson AM, Rich SN, Russo ME, Bhatnagar J, Kumar RN, Ritter JM, Annambhotla P, Takeda MR, Kuhn KF, Pillai P, DeLeon-Carnes M, Scobell R, Ekambaram M, Finkel R, Reagan-Steiner S, Martines RB, Satoskar RS, Vranic GM, Mohammed R, Rivera GE, Cooper K, Abdelal H, Couturier MR, Bradley BT, Hinckley AF, Koehler JE, Mead PS, Kuehnert MJ, Ackelsberg J, Basavaraju SV, Marx GE. Beeson AM, et al. Among authors: finkel r. Emerg Infect Dis. 2024 Dec;30(12):2467-2475. doi: 10.3201/eid3012.240310. Emerg Infect Dis. 2024. PMID: 39592261 Free article. Review.
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases.
Kim-McManus O, Gleeson JG, Mignon L, Smith Fine A, Yan W, Nolen N, Demarest S, Berry-Kravis E, Finkel R, Leonard S, Finlayson S, Augustine E, Lyon GJ, Schule R, Yu T. Kim-McManus O, et al. Among authors: finkel r. Nat Commun. 2024 Nov 12;15(1):9802. doi: 10.1038/s41467-024-54077-5. Nat Commun. 2024. PMID: 39532857 Free PMC article. Review.
Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.
Coratti G, Bovis F, Pera MC, Civitello M, Rohwer A, Salmin F, Glanzman AM, Montes J, Pasternak A, De Sanctis R, Dunaway Young S, Duong T, Mizzoni I, Milev E, Sframeli M, Morando S, Albamonte E, D'Amico A, Catteruccia M, Brolatti N, Pane M, Scoto M, Messina S, Escudero JE, De Waele L, Hirano M, Zolkipli-Cunningham Z, Darras BT, Bertini E, Nascimiento Osorio A, Bruno C, Goemans N, Sansone VA, Day J, Baranello G, Muntoni F, Finkel R, Mercuri E; ISMAC/international SMA consortium. Coratti G, et al. Among authors: finkel r. Eur J Neurol. 2024 Dec;31(12):e16517. doi: 10.1111/ene.16517. Epub 2024 Oct 11. Eur J Neurol. 2024. PMID: 39392101 Free PMC article.
N-of-1 Studies in an Era of Precision Medicine.
Augustine EF, Yu TW, Finkel RS. Augustine EF, et al. Among authors: finkel rs. JAMA. 2024 Sep 25. doi: 10.1001/jama.2024.14637. Online ahead of print. JAMA. 2024. PMID: 39320868
Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module.
Coratti G, Civitello M, Rohwer A, Albamonte E, Montes J, Glanzman AM, Pasternak A, De Sanctis R, Young SD, Duong T, Mizzoni I, Milev E, Sframeli M, Morando S, D'Amico A, Catteruccia M, Brolatti N, Pane M, Scoto M, Messina S, Hirano M, Zolkipli-Cunningham Z, Darras BT, Bertini E, Bruno C, Sansone VA, Salmin F, Day J, Baranello G, Pera MC, Muntoni F, Finkel RS, Mercuri E; International SMA Consortium (iSMAc). Coratti G, et al. Among authors: finkel rs. Neuromuscul Disord. 2024 Nov;44:104449. doi: 10.1016/j.nmd.2024.08.006. Epub 2024 Aug 28. Neuromuscul Disord. 2024. PMID: 39299818 Free article.
Reliability of In Vivo Creatine-Weighted Chemical Exchange Saturation Transfer (CrCEST) MRI in Calf Skeletal Muscle of Healthy Volunteers at 3 T.
Chakraborty K, Burman R, Nisar S, Miller S, Loschinskey Z, Wu S, Li Y, Bag AK, Khan A, Goodenough C, Wilson N, Haris M, McCormack SE, Reddy R, Ness K, Finkel R, Bagga P. Chakraborty K, et al. Among authors: finkel r. J Magn Reson Imaging. 2024 Aug 30. doi: 10.1002/jmri.29566. Online ahead of print. J Magn Reson Imaging. 2024. PMID: 39212126
326 results