E-LIB Suche - Ergebnisse für: Weinreb, Neal J.

1

Characteristics and management of US pediatric patients wit..:

Goker-Alpan, Ozlem ; Burrow, Andrew ; Enns, Gregory M....
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107854 , 2024

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

2

The international cooperative Gaucher group (ICCG) Gaucher ..:

Weinreb, Neal J.
Best Practice & Research Clinical Haematology. 36 (2023) 4 - p. 101522 , 2023

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

3

The Two Substrate Reduction Therapies for Type 1 Gaucher Di..:

Mistry, Pramod K. ; Kishnani, Priya S. ; Balwani, Manisha...
Journal of Clinical Medicine. 12 (2023) 9 - p. 3269 , 2023

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

4

P310: Project Searchlight study methodology: Real-world eva..:

King, Lisa Sniderman ; Aguiar, Mario ; Chiorean, Alexandra...
Genetics in Medicine Open. 1 (2023) 1 - p. 100338 , 2023

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

5

Project Searchlight Gaucher study design: Real-world evalua..:

King, Lisa Sniderman ; Aguiar, Mario ; Chiorean, Alexandra...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S114 , 2022

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

6

Screening, patient identification, evaluation, and treatmen..:

Kishnani, Priya S. ; Al-Hertani, Walla ; Balwani, Manisha...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. 154-162 , 2022

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

7

Development and validation of Gaucher disease type 1 (GD1)-..:

Elstein, Deborah ; Belmatoug, Nadia ; Deegan, Patrick...
Orphanet Journal of Rare Diseases. 17 (2022) 1 - p. , 2022

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

8

Cancer risk and gammopathies in 2123 adults with Gaucher di..:

Rosenbloom, Barry E. ; Cappellini, Maria Domenica ; Weinreb, Neal J....
American Journal of Hematology. 97 (2022) 10 - p. 1337-1347 , 2022

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

9

Plain Language Summary of the International Collaborative G..:

Deegan, Patrick ; Khan, Aneal ; Camelo Jr, José Simon.
Future Rare Diseases. 2 (2022) 4 - p. , 2022

Link: https://doi.org/10.2217/..

10

Gaucher disease – more than just a rare lipid storage disea..:

Roh, Jaehyeok ; Subramanian, Subbaya ; Weinreb, Neal J..
Journal of Molecular Medicine. 100 (2022) 4 - p. 499-518 , 2022

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

11

Safety and effectiveness of taliglucerase alfa in patients ..:

Titievsky, Lina ; Schuster, Tilman ; Wang, Ronnie...
Orphanet Journal of Rare Diseases. 17 (2022) 1 - p. , 2022

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

12

The diagnosis and management of Gaucher disease in pediatri..:

Weinreb, Neal J. ; Goker-Alpan, Ozlem ; Kishnani, Priya S....
Molecular Genetics and Metabolism. 136 (2022) 1 - p. 4-21 , 2022

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

13

Physiologically‐Based Pharmacokinetic Model Development, Va..:

Sahasrabudhe, Siddhee A. ; Cheng, Shen ; Al‐Kofahi, Mahmoud...
Clinical Pharmacology & Therapeutics. 112 (2022) 6 - p. 1254-1263 , 2022

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

14

Hematologic malignancies and monoclonal gammopathy of undet..:

Rosenbloom, Barry ; Cappellini, Maria D. ; Dragosky, Marta...
Molecular Genetics and Metabolism. 132 (2021) 2 - p. S91-S92 , 2021

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

15

COVID-19 patient impact: A survey of the Gaucher community ..:

Ryan, Emory ; Lopez, Grisel ; Balwani, Manisha...
Molecular Genetics and Metabolism. 132 (2021) 2 - p. S93 , 2021

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