Parent Project Muscular Dystrophy[Grants and Funding] - Search Results

Year	Number of Results
2018	2
2019	3
2021	2
2023	1
2024	10
2025	0

1

Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.

Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR. Mendell JR, et al. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.

2

Patient- and caregiver-reported impact of symptoms in Duchenne muscular dystrophy.

Rosero S, Weinstein J, Seabury J, Varma A, Dilek N, Zizzi C, Coffey M, Greco B, Heatwole J, Alexandrou D, Guntrum D, Ciafaloni E, Heatwole C. Rosero S, et al. Muscle Nerve. 2024 Jul;70(1):120-129. doi: 10.1002/mus.28102. Epub 2024 May 8. Muscle Nerve. 2024. PMID: 38720616

3

Cardiac atrial pathology in Duchenne muscular dystrophy.

Greiner E, Breaux A, Kasten J, Seo J, Ollberding NJ, Spar D, Ryan TD, Lang SM, Tian C, Sawnani H, Villa CR. Greiner E, et al. Muscle Nerve. 2024 May;69(5):572-579. doi: 10.1002/mus.28072. Epub 2024 Mar 1. Muscle Nerve. 2024. PMID: 38426616

4

Risk Factors Associated with Incident Vertebral Fractures in Steroid-treated Males with Duchenne Muscular Dystrophy.

Phung K, McAdam L, Ma J, McMillan HJ, Jackowski S, Scharke M, Matzinger MA, Shenouda N, Koujok K, Jaremko JL, Wilson N, Walker S, Hartigan C, Khan N, Page M, Robinson ME, Saleh DS, Smit K, Rauch F, Siminoski K, Ward LM. Phung K, et al. J Clin Endocrinol Metab. 2024 Jan 18;109(2):536-548. doi: 10.1210/clinem/dgad435. J Clin Endocrinol Metab. 2024. PMID: 37610420

5

Reductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years.

Evans WJ, Hellerstein M, Butterfield RJ, Smith E, Guglieri M, Katz N, Nave B, Branigan L, Thera S, Vordos KL, Behar L, Schiava M, James MK, Field T, Mohammed H, Shankaran M. Evans WJ, et al. J Physiol. 2024 Oct;602(19):4929-4939. doi: 10.1113/JP287069. Epub 2024 Aug 31. J Physiol. 2024. PMID: 39216089

6

Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy.

McDonald CM. McDonald CM. Muscle Nerve. 2018 Nov;58(5):614-617. doi: 10.1002/mus.26334. Epub 2018 Oct 19. Muscle Nerve. 2018. PMID: 30192014 No abstract available.

7

Assessing the Benefits and Harms Associated with Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne Muscular Dystrophy.

Bhattacharyya O, Campoamor NB, Armstrong N, Freed M, Schrader R, Crossnohere NL, Bridges JFP. Bhattacharyya O, et al. Int J Neonatal Screen. 2024 Apr 15;10(2):32. doi: 10.3390/ijns10020032. Int J Neonatal Screen. 2024. PMID: 38651397 Free PMC article.

8

Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals.

Crossnohere NL, Campoamor NB, Camino E, Dresnick E, Martschenko DO, Rodrigues V, Apkon S, Hazlett A, Mittur D, Rodriguez PE, Bridges JFP, Armstrong N. Crossnohere NL, et al. Orphanet J Rare Dis. 2024 May 21;19(1):207. doi: 10.1186/s13023-024-03209-7. Orphanet J Rare Dis. 2024. PMID: 38773664 Free PMC article.

9

Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy.

Hollin IL, Peay H, Fischer R, Janssen EM, Bridges JFP. Hollin IL, et al. Qual Life Res. 2018 Sep;27(9):2261-2273. doi: 10.1007/s11136-018-1891-7. Epub 2018 May 26. Qual Life Res. 2018. PMID: 29804169

10

Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry.

Cowen L, Mancini M, Martin A, Lucas A, Donovan JM. Cowen L, et al. BMC Neurol. 2019 May 2;19(1):84. doi: 10.1186/s12883-019-1304-8. BMC Neurol. 2019. PMID: 31046703 Free PMC article.

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