Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

122 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Long-Term Follow-Up Cares and Check Initiative: A Program to Advance Long-Term Follow-Up in Newborns Identified with a Disease through Newborn Screening.
Lietsch M, Chan K, Taylor J, Lee BH, Ciafaloni E, Kwon JM, Waldrop MA, Butterfield RJ, Rathore G, Veerapandiyan A, Kapil A, Parsons JA, Gibbons M, Brower A. Lietsch M, et al. Among authors: butterfield rj. Int J Neonatal Screen. 2024 Apr 18;10(2):34. doi: 10.3390/ijns10020034. Int J Neonatal Screen. 2024. PMID: 38651399 Free PMC article.
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley G, Johnson K, Foster R, Gheuens S, Bhan I, Reyna SP, Fradette S, Farwell W; NURTURE Study Group. De Vivo DC, et al. Among authors: butterfield rj. Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12. Neuromuscul Disord. 2019. PMID: 31704158 Free PMC article. Clinical Trial.
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.
Veerapandiyan A, Wagner KR, Apkon S, McDonald CM, Mathews KD, Parsons JA, Wong BL, Eichinger K, Shieh PB, Butterfield RJ, Rao VK, Smith EC, Proud CM, Connolly AM, Ciafaloni E. Veerapandiyan A, et al. Among authors: butterfield rj. Muscle Nerve. 2020 Jul;62(1):41-45. doi: 10.1002/mus.26902. Epub 2020 May 5. Muscle Nerve. 2020. PMID: 32329920 Free PMC article. Review.
Spinal muscular atrophy care in the COVID-19 pandemic era.
Veerapandiyan A, Connolly AM, Finkel RS, Arya K, Mathews KD, Smith EC, Castro D, Butterfield RJ, Parsons JA, Servais L, Kuntz N, Rao VK, Brandsema JF, Mercuri E, Ciafaloni E. Veerapandiyan A, et al. Among authors: butterfield rj. Muscle Nerve. 2020 Jul;62(1):46-49. doi: 10.1002/mus.26903. Epub 2020 May 3. Muscle Nerve. 2020. PMID: 32329921 Free PMC article. Review.
Medical management of muscle weakness in Duchenne muscular dystrophy.
Rivera SR, Jhamb SK, Abdel-Hamid HZ, Acsadi G, Brandsema J, Ciafaloni E, Darras BT, Iannaccone ST, Konersman CG, Kuntz NL, McDonald CM, Parsons JA, Tesi Rocha C, Zaidman CM, Butterfield RJ, Connolly AM, Mathews KD. Rivera SR, et al. Among authors: butterfield rj. PLoS One. 2020 Oct 19;15(10):e0240687. doi: 10.1371/journal.pone.0240687. eCollection 2020. PLoS One. 2020. PMID: 33075081 Free PMC article.
Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet).
Mathews KD, Conway KM, Gedlinske AM, Johnson N, Street N, Butterfield RJ, Hung M, Ciafaloni E, Romitti PA. Mathews KD, et al. Among authors: butterfield rj. Children (Basel). 2021 Sep 23;8(10):835. doi: 10.3390/children8100835. Children (Basel). 2021. PMID: 34682100 Free PMC article.
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.
Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Waldrop MA, et al. Among authors: butterfield rj. Hum Mutat. 2022 Apr;43(4):511-528. doi: 10.1002/humu.24343. Epub 2022 Mar 7. Hum Mutat. 2022. PMID: 35165973 Free PMC article.
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ Jr, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M. Crow RA, et al. Among authors: butterfield rj. Trials. 2018 May 10;19(1):291. doi: 10.1186/s13063-018-2645-0. Trials. 2018. PMID: 29793540 Free PMC article. Review.
Association of genetic mutations and loss of ambulation in childhood-onset dystrophinopathy.
Haber G, Conway KM, Paramsothy P, Roy A, Rogers H, Ling X, Kozauer N, Street N, Romitti PA, Fox DJ, Phan HC, Matthews D, Ciafaloni E, Oleszek J, James KA, Galindo M, Whitehead N, Johnson N, Butterfield RJ, Pandya S, Venkatesh S, Bhattaram VA. Haber G, et al. Among authors: butterfield rj. Muscle Nerve. 2021 Feb;63(2):181-191. doi: 10.1002/mus.27113. Epub 2020 Nov 17. Muscle Nerve. 2021. PMID: 33150975 Free PMC article.
122 results