E-LIB Suche - Ergebnisse für: Kennedy, Annie

1

The Early Care (0–3 Years) In Duchenne Muscular Dystrophy M..:

Armstrong, Niki ; Apkon, Susan ; Berggren, Kiera N....
Journal of Neuromuscular Diseases. 11 (2024) 2 - p. 525-533 , 2024

Link: https://doi.org/10.3233/..

2

Circulating tumor DNA reveals mechanisms of lorlatinib resi..:

Berko, Esther R. ; Witek, Gabriela M. ; Matkar, Smita...
Nature Communications. 14 (2023) 1 - p. , 2023

Link: https://doi.org/10.1038/..

3

A Foundation for Patient-Centered Core Impact Sets: Key Lea..:

Perfetto, Eleanor M. ; Love, T. Rosie ; Oehrlein, Elisabeth M....
The Patient - Patient-Centered Outcomes Research. 16 (2023) 4 - p. 293-300 , 2023

Link: https://doi.org/10.1007/..

4

Are we prepared to deliver gene‐targeted therapies for rare..:

Yu, Timothy W. ; Kingsmore, Stephen F. ; Green, Robert C....
American Journal of Medical Genetics Part C: Seminars in Medical Genetics. 193 (2023) 1 - p. 7-12 , 2023

Link: https://doi.org/10.1002/..

5

Newborn screening for Duchenne muscular dystrophy: A two‐ye..:

Tavakoli, Norma P. ; Gruber, Dorota ; Armstrong, Niki...
Annals of Clinical and Translational Neurology. 10 (2023) 8 - p. 1383-1396 , 2023

Link: https://doi.org/10.1002/..

6

Patient-Centered Core Impact Sets: What They are and Why We..:

Perfetto, Eleanor M. ; Oehrlein, Elisabeth M. ; Love, T. Rosie...
The Patient - Patient-Centered Outcomes Research. 15 (2022) 6 - p. 619-627 , 2022

Link: https://doi.org/10.1007/..

7

Common Challenges and Identified Solutions for State Newbor..:

Simon, Dylan ; Broadbridge, Elizabeth ; Baker, Mei...
International Journal of Neonatal Screening. 8 (2022) 1 - p. 7 , 2022

Link: https://doi.org/10.3390/..

8

A review of common data elements in RUSP submission package:

Broadbridge, Liesl ; Simon, Dylan ; Sullivan, Jamie.
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S26 , 2022

Link: https://doi.org/10.1016/..

9

The national economic burden of rare disease in the United ..:

Yang, Grace ; Cintina, Inna ; Pariser, Anne...
Orphanet Journal of Rare Diseases. 17 (2022) 1 - p. , 2022

Link: https://doi.org/10.1186/..

10

Correction: Data Sharing Goals for Nonprofit Funders of Cli..:

Coetzee, Timothy ; Ball, Mad Price ; Boutin, Marc...
Journal of Participatory Medicine. 13 (2021) 2 - p. e31371 , 2021

Link: https://doi.org/10.2196/..

11

The Evolving Regulatory Space and the Advent of Patient-Foc..:

, In: Rare Disease Drug Development,

Valentine, James E. ; Kennedy, Annie - p. 277-289 , 2021

Link: https://doi.org/10.1007/..

12

Data Sharing Goals for Nonprofit Funders of Clinical Trials:

Coetzee, Timothy ; Ball, Mad Price ; Boutin, Marc...
Journal of Participatory Medicine. 13 (2021) 1 - p. e23011 , 2021

Link: https://doi.org/10.2196/..

13

Stakeholder-Engaged Derivation of Patient-Informed Value El..:

dosReis, Susan ; Butler, Beverly ; Caicedo, Juan...
The Patient - Patient-Centered Outcomes Research. 13 (2020) 5 - p. 611-621 , 2020

Link: https://doi.org/10.1007/..

14

The sixth international RASopathies symposium: Precision me..:

Gripp, Karen W. ; Schill, Lisa ; Schoyer, Lisa...
American Journal of Medical Genetics Part A. 182 (2019) 3 - p. 597-606 , 2019

Link: https://doi.org/10.1002/..

15

Duchenne Muscular Dystrophy Newborn Screening: Evaluation o..:

Timonen, Anne ; Lloyd-Puryear, Michele ; Hougaard, David M....
International Journal of Neonatal Screening. 5 (2019) 3 - p. 27 , 2019

Link: https://doi.org/10.3390/..