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Page 1
Spinal Muscular Atrophy.
Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Nicolau S, et al. Among authors: connolly am. Semin Pediatr Neurol. 2021 Apr;37:100878. doi: 10.1016/j.spen.2021.100878. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892848 Review.
These agents have demonstrated safety and efficacy, but their long-term benefits require further study. Newborn screening programs are enabling earlier diagnosis and treatment and better outcomes, but respiratory care and other supportive measures retain a key role in the …
These agents have demonstrated safety and efficacy, but their long-term benefits require further study. Newborn screening programs ar …
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Clemens PR, et al. Among authors: connolly am. J Neuromuscul Dis. 2022;9(4):493-501. doi: 10.3233/JND-220811. J Neuromuscul Dis. 2022. PMID: 35634851 Free PMC article. Clinical Trial.
OBJECTIVE: To evaluate long-term efficacy and safety of the anti-sense oligonucleotide viltolarsen in the treatment of patients with DMD amenable to exon 53 skipping therapy. ...Both groups were treated with glucocorticoids. All 16 participants elected to enroll in this lo …
OBJECTIVE: To evaluate long-term efficacy and safety of the anti-sense oligonucleotide viltolarsen in the treatment of patients with …
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Mah JK, et al. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. JAMA Netw Open. 2022. PMID: 35076703 Free PMC article. Clinical Trial.
INTERVENTIONS: Participants were enrolled in a 24-month, long-term extension (LTE) study with vamorolone dose escalated to 2.0 or 6.0 mg/kg/d. ...
INTERVENTIONS: Participants were enrolled in a 24-month, long-term extension (LTE) study with vamorolone dose escalated to 2.0 or 6.0 …
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Clemens PR, et al. Among authors: connolly am. J Neuromuscul Dis. 2023;10(3):439-447. doi: 10.3233/JND-221656. J Neuromuscul Dis. 2023. PMID: 37005891 Free PMC article. Clinical Trial.
OBJECTIVE: To evaluate the efficacy and safety of viltolarsen for an additional 192 weeks in boys with DMD. METHODS: This phase 2, open-label, 192-week long-term extension (LTE) study (NCT03167255) evaluated the efficacy and safety of viltolarsen in participants aged 4 to …
OBJECTIVE: To evaluate the efficacy and safety of viltolarsen for an additional 192 weeks in boys with DMD. METHODS: This phase 2, open-labe …
Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.
McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators. McDonald CM, et al. Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. Epub 2017 Nov 22. Lancet. 2018. PMID: 29174484 Clinical Trial.
BACKGROUND: Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed the long-term benefits of this treatment. We examined the long-term effects of glucocorticoids on milestone-related disease p …
BACKGROUND: Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed …
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio.
Waldrop MA, Chagat S, Storey M, Meyer A, Iammarino M, Reash N, Alfano L, Lowes L, Noritz G, Prochoroff A, Rossman I, Ginsberg M, Mosher K, Broomall E, Bass N, Gushue C, Kotha K, Paul G, Shell R, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: connolly am. Neuromuscul Disord. 2024 Jan;34:41-48. doi: 10.1016/j.nmd.2023.11.010. Epub 2023 Dec 2. Neuromuscul Disord. 2024. PMID: 38142474
Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne Muscular Dystrophy: Clinical Practice Recommendations.
Bowden SA, Connolly AM, Kinnett K, Zeitler PS. Bowden SA, et al. Among authors: connolly am. J Neuromuscul Dis. 2019;6(1):31-41. doi: 10.3233/JND-180346. J Neuromuscul Dis. 2019. PMID: 30614808 Free PMC article. Review.
Long-term glucocorticoid therapy has improved outcomes in patients with Duchenne muscular dystrophy. ...
Long-term glucocorticoid therapy has improved outcomes in patients with Duchenne muscular dystrophy. ...
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.
Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N; investigators from the LNMRC Natural History study; McDonald CM; investigators from the CINRG Duchenne National History Study; Mercuri E; investigators from The DMD Italian Group; Mendell JR. Mitelman O, et al. Among authors: connolly am. J Neuromuscul Dis. 2022;9(1):39-52. doi: 10.3233/JND-210665. J Neuromuscul Dis. 2022. PMID: 34420980 Free PMC article. Clinical Trial.
Chart review Study 4658-405 (405) further followed these patients while receiving eteplirsen during usual clinical care. OBJECTIVE: To compare long-term clinical outcomes of eteplirsen-treated patients from Studies 201/202/405 with those of external controls. ...
Chart review Study 4658-405 (405) further followed these patients while receiving eteplirsen during usual clinical care. OBJECTIVE: To compa …
Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy.
Meyer AP, Connolly AM, Vannatta K, Hacker N, Hatfield A, Decipeda A, Parker P, Willoughby A, Waldrop MA. Meyer AP, et al. Among authors: connolly am. J Neuromuscul Dis. 2024;11(1):129-142. doi: 10.3233/JND-230082. J Neuromuscul Dis. 2024. PMID: 38160362 Free PMC article.
RESULTS: Gene replacement therapy was most parents' first treatment choice, although concerns regarding long term efficacy (65%) and safety (51%) were common. Information provided during the newborn screening disclosure was quite variable. ...
RESULTS: Gene replacement therapy was most parents' first treatment choice, although concerns regarding long term efficacy (65%) and …
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy.
Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Ke Q, et al. Among authors: connolly am. World J Pediatr. 2019 Jun;15(3):219-225. doi: 10.1007/s12519-019-00242-6. Epub 2019 Mar 23. World J Pediatr. 2019. PMID: 30904991 Review.
CONCLUSIONS: Approved treatments for DMD and SMA may change the natural history of these diseases. Long-term studies of these treatments are underway. To avoid the known diagnostic delay associated with these disorders and provide optimal effectiveness of these treatments, …
CONCLUSIONS: Approved treatments for DMD and SMA may change the natural history of these diseases. Long-term studies of these treatme …
24 results