Al‐Zaidy, Samiah: search on Z-Library

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1

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Na

Journal:

New England Journal of Medicine

Year:

2017

Language:

english

File:

PDF, 338 KB

english, 2017

2

Milder phenotype of congenital muscular dystrophy in a novel POMT1 mutation

Samiah A. Al-Zaidy, Berivan Baskin, Cynthia Hawkins, Grace Yoon, Peter N. Ray, Jiri Vajsar

Journal:

Year:

2012

Language:

english

File:

PDF, 1.83 MB

english, 2012

3

DUCHENNE AND BECKER MUSCULAR DYSTROPHY CARRIERS: EVIDENCE OF CARDIOMYOPATHY BY EXERCISE AND CARDIAC MAGNETIC RESONANCE IMAGING

Mah, May Ling, Cripe, Linda, Slawinski, Michelle, Camino, Eric, Al-Zaidy, Samiah, Lehman, Kelly, Jackson, Jamie, Mendell, Jerry, Hor, Kan

Journal:

Journal of the American College of Cardiology

Year:

2019

Language:

english

File:

PDF, 173 KB

english, 2019

4

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

Al-Zaidy, Samiah, Rodino-Klapac, Louise, Mendell, Jerry R.

Journal:

Pediatric Neurology

Year:

2014

Language:

english

File:

PDF, 2.90 MB

english, 2014

5

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones

Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. David, Rodino-Klapac, Louise, Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nage

Journal:

European Journal of Paediatric Neurology

Year:

2017

Language:

english

File:

PDF, 169 KB

english, 2017

6

Neurological Complications of PCR-Proven M. pneumoniae Infections in Children: Prodromal Illness Duration May Reflect Pathogenetic Mechanism

Al-Zaidy, Samiah A., MacGregor, Daune, Mahant, Sanjay, Richardson, Susan E., Bitnun, Ari

Journal:

Clinical Infectious Diseases

Year:

2015

Language:

english

File:

PDF, 1012 KB

english, 2015

7

480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W Dave, Rodino-Klapac, Louise, Kissel, John T., Prior, Thomas W., Miranda, Carlos, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Petek,

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 494 KB

english, 2016

8

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Al-Zaidy, Samiah A., Kolb, Stephen J., Lowes, Linda, Alfano, Lindsay N., Shell, Richard, Church, Kathleen R., Nagendran, Sukumar, Sproule, Douglas M., Feltner, Douglas E., Wells, Courtney, Ogrinc, Fra

Journal:

Journal of Neuromuscular Diseases

Year:

2019

Language:

english

File:

PDF, 315 KB

english, 2019

9

DUCHENNE AND BECKER MUSCULAR DYSTROPHY CARRIERS: EVIDENCE OF CARDIOMYOPATHY BY EXERCISE AND CARDIAC MAGNETIC RESONANCE IMAGING

Mah, May Ling, Cripe, Linda, Slawinski, Michelle, Camino, Eric, Al-Zaidy, Samiah, Lehman, Kelly, Jackson, Jamie, Mendell, Jerry, Hor, Kan

Journal:

Journal of the American College of Cardiology

Year:

2019

File:

PDF, 173 KB

2019

10

Clinical Follow-Up For Duchenne Muscular Dystrophy Newborn Screening: A Proposal

Kwon, Jennifer M., Abdel-Hamid, Hoda Z., Al-Zaidy, Samiah A., Mendell, Jerry R., Kennedy, Annie, Kinnett, Kathi, Cwik, Valerie A., Street, Natalie, Bolen, Julie, Day, John W., Connolly, Anne M.

Journal:

Year:

2016

Language:

english

File:

PDF, 149 KB

english, 2016

11

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine,

Journal:

Pediatric Pulmonology

Year:

2018

Language:

english

File:

PDF, 443 KB

english, 2018

12

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P., Miller, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church,

Journal:

Year:

2020

File:

PDF, 1.60 MB

2020

13

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D (LGMD2D)

Mendell, Jerry R., Chicoine, Louis G, Al-Zaidy, Samiah A, Sahenk, Zarife, Lehman, Kelly, Lowes, Linda, Miller, Natalie, Alfano, Lindsay, Galliers, Beverly, Lewis, Sarah, Murrey, Darren, Peterson, Elly

Journal:

Human Gene Therapy

Year:

2019

Language:

english

File:

PDF, 971 KB

english, 2019

14

Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing

Mah, May Ling, Cripe, Linda, Slawinski, Michelle K., Al-Zaidy, Samiah A., Camino, Eric, Lehman, Kelly J., Jackson, Jamie L., Iammarino, Megan, Miller, Natalie, Mendell, Jerry R., Hor, Kan N.

Journal:

International Journal of Cardiology

Year:

2020

File:

PDF, 901 KB

2020

15

A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican Hispanics

Al-Zaidy, Samiah A., Malik, Vinod, Kneile, Kelley, Rosales, Xiomara Q., Gomez, Ana Maria, Lewis, Sarah, Hashimoto, Sayaka, Gastier-Foster, Julie, Kang, Peter, Darras, Basil, Kunkel, Louis, Carlo, Jose

Journal:

Molecular Genetics & Genomic Medicine

Year:

2015

Language:

english

File:

PDF, 2.03 MB

english, 2015

16

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel

Journal:

Molecular Therapy

Year:

2017

Language:

english

File:

PDF, 1.27 MB

english, 2017

17

A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy

Al-Zaidy, Samiah, Lloyd-Puryear, Michele, Kennedy, Annie, Lopez, Veronica, Mendell, Jerry R.

Journal:

International Journal of Neonatal Screening

Year:

2017

Language:

english

File:

PDF, 1.23 MB

english, 2017

18

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

Al-Zaidy, Samiah A., Sahenk, Zarife, Rodino-Klapac, Louise R., Kaspar, Brian, Mendell, Jerry R.

Journal:

Journal of Neuromuscular Diseases

Year:

2015

Language:

english

File:

PDF, 534 KB

english, 2015

19

497. Follistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM)

Mendell, Jerry R., Sahenk, Zarife, Hogan, Mark, Al-Zaidy, Samiah, Flanigan, Kevin, Rodino-Klapac, Louise R., McColly, Markus, Church, Kathleen, Lewis, Sarah, Lowes, Linda, Alfano, Lindsay, Berry, Kath

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 1.02 MB

english, 2016

20

Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age GroupâMatched Controls

Jackson, Jamie L., Korth, Christina X., Leslie, Carine E., Cotto, Jennifer, Mah, May Ling, Hor, Kan, Cripe, Linda, Al-Zaidy, Samiah, Camino, Eric M., Church, Kathleen, Lehman, Kelly J., Shay, Victoria

Journal:

Journal of Child Neurology

Year:

2020

File:

PDF, 184 KB

2020

21

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use

Shell, Richard, Al-Zaidy, Samiah, Arnold, W. Dave, Rodino-Klapac, Louise, Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L'It

Journal:

European Journal of Paediatric Neurology

Year:

2017

Language:

english

File:

PDF, 43 KB

english, 2017

22

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

Al-Zaidy, Samiah A., Mendell, Jerry R.

Journal:

Pediatric Neurology

Year:

2019

Language:

english

File:

PDF, 451 KB

english, 2019

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