|
🐜
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Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
15 auth.
M. Passini,
J. Bu,
A. Richards,
Cathrine Kinnecom,
S. Sardi,
L. Stanek,
Y. Hua,
F. Rigo,
J. Matson,
G. Hung,
...
E. Kaye,
L. Shihabuddin,
A. Krainer,
C. Bennett,
Seng H. Cheng
|
8 |
2011 |
8
🐜
|
|
🐜
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Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
39 auth.
J. Mendell,
N. Goemans,
L. Lowes,
L. Alfano,
K. Berry,
James Shao,
E. Kaye,
E. Mercuri,
H. Hamid,
B. Byrne,
A. Connolly,
R. Dracker,
L. Matthew Frank,
P. Heydemann,
K. O'Brien,
...
S. Sparks,
L. Specht,
L. Rodino-Klapac,
Z. Sahenk,
S. Al-Zaidy,
L. Cripe,
S. Lewis,
P. M,
Mazzone E,
M. S,
Vita Gl,
D. Bertini,
Berardinelli A Casimiro,
Torrente Y,
M. F.,
Comi Gp,
Baranello G,
M. T,
Pini A,
Battini R,
Pegoraro E,
Bruno C,
P. L.,
Previtali S
|
8 |
2015 |
8
🐜
|
|
🐢
|
Eteplirsen for the treatment of Duchenne muscular dystrophy
22 auth.
J. Mendell,
L. Rodino-Klapac,
Z. Sahenk,
K. Roush,
L. Bird,
L. Lowes,
L. Alfano,
A. M. Gomez,
S. Lewis,
Janaiah Kota,
...
V. Malik,
K. Shontz,
Christopher M. Walker,
K. Flanigan,
Marco Corridore,
J. Kean,
Hugh D. Allen,
C. Shilling,
K. Melia,
P. Sazani,
J. Saoud,
E. Kaye
|
8 |
2013 |
8
🐢
|
|
🐜
|
Clinical research for rare disease: opportunities, challenges, and solutions.
9 auth.
R. Griggs,
M. Batshaw,
M. Dunkle,
R. Gopal‐srivastava,
E. Kaye,
J. Krischer,
...
Tan Nguyen,
Kathleen Paulus,
P. Merkel
|
8 |
2009 |
8
🐜
|
|
🐜
|
Childhood ataxia with diffuse central nervous system hypomyelination
15 auth.
R. Schiffmann,
J. Moller,
B. Trapp,
H. H. Shih,
R. Farrer,
D. Katz,
J. Alger,
C. Parker,
Peter E. Hauer,
C. Kaneski,
...
John D Heiss,
E. Kaye,
R. Quarles,
R. Brady,
N. Barton
|
8 |
1994 |
8
🐜
|
|
🐜
|
A CONTROLLED TRIAL OF DIAZEPAM ADMINISTERED DURING FEBRILE ILLNESSES TO PREVENT RECURRENCE OF FEBRILE SEIZURES
8 auth.
N. Rosman,
Theodore Colton,
Jan L. Labazzo,
P. L. Gilbert,
Nancy B. Gardella,
E. Kaye,
...
C. V. Van Bennekom,
M. R. Winter
|
7 |
1993 |
7
🐜
|
|
🐢
|
MR diffusion imaging and MR spectroscopy of maple syrup urine disease during acute metabolic decompensation
W. Jan,
R. Zimmerman,
Zhiyue J. Wang,
G. Berry,
P. Kaplan,
E. Kaye
|
7 |
2003 |
7
🐢
|
|
🦁
|
Nervous system involvement in Fabry's disease: Clinicopathological and biochemical correlation
E. Kaye,
E. Kolodny,
E. Logigian,
M. Ullman
|
7 |
1988 |
7
🦁
|
|
🐜
|
Newborn screening by tandem mass spectrometry for medium-chain Acyl-CoA dehydrogenase deficiency: a cost-effectiveness analysis.
7 auth.
L. Venditti,
C. Venditti,
G. Berry,
P. Kaplan,
E. Kaye,
H. Glick,
...
C. Stanley
|
6 |
2003 |
6
🐜
|
|
🦁
|
Update on genetic disorders affecting white matter.
E. Kaye
|
6 |
2001 |
6
🦁
|
|
🦁
|
Type 2 and type 3 Gaucher disease: A morphological and biochemical study
E. Kaye,
M. Ullman,
E. Wilson,
J. Barranger
|
6 |
1986 |
6
🦁
|
