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Author/Creator Balwani, Manisha

2. Acute Intermittent Porphyria: Predicted Pathogenicity of HMBS Variants Indicates Extremely Low Penetrance of the Autosomal Dominant Disease. Issue 11 (5th September 2016)

Authors:
Chen, Brenden; Solis‐Villa, Constanza; Hakenberg, Jörg; Qiao, Wanqiong; Srinivasan, Ramakrishnan R.; Yasuda, Makiko; Balwani, Manisha; Doheny, Dana; Peter, Inga; Chen, Rong; Desnick, Robert J.
Journal:
Human mutation
Issue:
Volume 37:Issue 11(2016)
Page Start:
1215
Record Type:
Journal Article
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3. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease. Issue 3 (28th March 2013)

Authors:
Balwani, Manisha; Breen, Catherine; Enns, Gregory M.; Deegan, Patrick B.; Honzík, Tomas; Jones, Simon; Kane, John P.; Malinova, Vera; Sharma, Reena; Stock, Eveline O.; Valayannopoulos, Vassili; Wraith, J. Edmond; Burg, Jennifer; Eckert, Stephen; Schneider, Eugene; Quinn, Anthony G.
Journal:
Hepatology
Issue:
Volume 58:Issue 3(2013:Sep.)
Page Start:
950
Record Type:
Journal Article
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4. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. Issue 9 (11th July 2021)

Authors:
Mistry, Pramod K.; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma P.; Baris Feldman, Hagit; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Lau, Heather; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Foster, Meredith C.; Gaemers, Sebastiaan...
Journal:
American journal of hematology
Issue:
Volume 96:Issue 9(2021)
Page Start:
1156
Record Type:
Journal Article
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6. Disease Characteristics of Acute Hepatic Porphyria Patients: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double‐Blind, Placebo‐Controlled Trial: 996. (October 2019)

Authors:
Anderson, Karl; Balwani, Manisha; Sardh, Eliane; Gouya, Laurent; Rees, David; Stein, Penelope; Stolzel, Ulrich; Aguilera, Paula; Bissell, Montgomery D.; Bonkovsky, Herbert; Keel, Sioban; Parker, Charles; Phillips, John; Silver, Samuel; Windyga, Jerzy; D'Avola, Delia; Ross, Gayle; Stewart, Peter; ...
Journal:
American journal of gastroenterology
Issue:
Volume 114 (2019)ACG Abstracts 1:Supplement
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Record Type:
Journal Article
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7. Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study. (16th November 2021)

Authors:
Ventura, Paolo; Bonkovsky, Herbert L.; Gouya, Laurent; Aguilera‐Peiró, Paula; Montgomery Bissell, D.; Stein, Penelope E.; Balwani, Manisha; Anderson, D. Karl E.; Parker, Charles; Kuter, David J.; Monroy, Susana; Oh, Jeeyoung; Ritchie, Bruce; Ko, John J.; Hua, Zhaowei; Sweetser, Marianne T.; Sardh...
Journal:
Liver international
Issue:
Volume 42:Number 1(2022)
Page Start:
161
Record Type:
Journal Article
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8. Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial. Issue 9985 (13th June 2015)

Authors:
Cox, Timothy M; Drelichman, Guillermo; Cravo, Renata; Balwani, Manisha; Burrow, Thomas Andrew; Martins, Ana Maria; Lukina, Elena; Rosenbloom, Barry; Ross, Leorah; Angell, Jennifer; Puga, Ana Cristina
Journal:
Lancet
Issue:
Volume 385:Issue 9985(2015)
Page Start:
2355
Record Type:
Journal Article
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9. Evaluating quality of life tools in North American patients with erythropoietic protoporphyria and X‐linked protoporphyria. Issue 1 (14th September 2019)

Authors:
Naik, Hetanshi; Overbey, Jessica R.; Desnick, Robert J.; Anderson, Karl E.; Bissell, D. Montgomery; Bloomer, Joseph; Bonkovsky, Herbert L.; Phillips, John D.; Wang, Bruce; Singal, Ashwani; Balwani, Manisha
Journal:
JIMD reports
Issue:
Volume 50:Issue 1(2019)
Page Start:
9
Record Type:
Journal Article
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10. EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks. Issue 5 (7th November 2019)

Authors:
Gouya, Laurent; Ventura, Paolo; Balwani, Manisha; Bissell, D. Montgomery; Rees, David C.; Stölzel, Ulrich; Phillips, John D.; Kauppinen, Raili; Langendonk, Janneke G.; Desnick, Robert J.; Deybach, Jean‐Charles; Bonkovsky, Herbert L.; Parker, Charles; Naik, Hetanshi; Badminton, Michael; Stein, Pen...
Journal:
Hepatology
Issue:
Volume 71:Issue 5(2020)
Page Start:
1546
Record Type:
Journal Article
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