Literature DB >> 35576532

Lessons Learned From the Long-Term Use of Enzyme Replacement Therapy in the Treatment of Lysosomal Acid Lipase Deficiency.

Alanna Strong1,2, Can Ficicioglu1,3.   

Abstract

Entities:  

Mesh：

Substances：

Year:  2022        PMID： 35576532      PMCID： PMC9296543          DOI： 10.1097/MPG.0000000000003453

Source DB:  PubMed          Journal:  J Pediatr Gastroenterol Nutr        ISSN： 0277-2116            Impact factor:   3.288


× No keyword cloud information.
  11 in total

Review 1.  Lysosomal Acid Lipase Deficiency: Therapeutic Options.

Authors:  Gregory M Pastores; Derralynn A Hughes
Journal:  Drug Des Devel Ther       Date:  2020-02-11       Impact factor: 4.162

Review 2.  Genetics and epigenetics of NAFLD and NASH: Clinical impact.

Authors:  Mohammed Eslam; Luca Valenti; Stefano Romeo
Journal:  J Hepatol       Date:  2017-11-06       Impact factor: 25.083

3.  Role of lysosomal acid lipase in the metabolism of plasma low density lipoprotein. Observations in cultured fibroblasts from a patient with cholesteryl ester storage disease.

Authors:  J L Goldstein; S E Dana; J R Faust; A L Beaudet; M S Brown
Journal:  J Biol Chem       Date:  1975-11-10       Impact factor: 5.157

4.  Long-Term Sebelipase Alfa Treatment in Children and Adults With Lysosomal Acid Lipase Deficiency.

Authors:  Barbara K Burton; Alejandra Consuelo Sanchez; Maria Kostyleva; Ana Maria Martins; Sachin Marulkar; Florian Abel; Ivo Barić
Journal:  J Pediatr Gastroenterol Nutr       Date:  2022-04-19       Impact factor: 3.288

Review 5.  Cholesteryl ester storage disease: review of the findings in 135 reported patients with an underdiagnosed disease.

Authors:  Donna L Bernstein; Helena Hülkova; Martin G Bialer; Robert J Desnick
Journal:  J Hepatol       Date:  2013-02-26       Impact factor: 25.083

6.  Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency.

Authors:  Vassili Valayannopoulos; Vera Malinova; Tomas Honzík; Manisha Balwani; Catherine Breen; Patrick B Deegan; Gregory M Enns; Simon A Jones; John P Kane; Eveline O Stock; Radhika Tripuraneni; Stephen Eckert; Eugene Schneider; Gavin Hamilton; Michael S Middleton; Claude Sirlin; Bruce Kessler; Christopher Bourdon; Simeon A Boyadjiev; Reena Sharma; Chris Twelves; Chester B Whitley; Anthony G Quinn
Journal:  J Hepatol       Date:  2014-06-30       Impact factor: 25.083

Review 7.  Determinants of fibrosis progression and regression in NASH.

Authors:  Detlef Schuppan; Rambabu Surabattula; Xiao Yu Wang
Journal:  J Hepatol       Date:  2017-11-14       Impact factor: 25.083

8.  A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.

Authors:  Barbara K Burton; Manisha Balwani; François Feillet; Ivo Barić; T Andrew Burrow; Carmen Camarena Grande; Mahmut Coker; Alejandra Consuelo-Sánchez; Patrick Deegan; Maja Di Rocco; Gregory M Enns; Richard Erbe; Fatih Ezgu; Can Ficicioglu; Katryn N Furuya; John Kane; Christina Laukaitis; Eugen Mengel; Edward G Neilan; Scott Nightingale; Heidi Peters; Maurizio Scarpa; K Otfried Schwab; Vratislav Smolka; Vassili Valayannopoulos; Marnie Wood; Zachary Goodman; Yijun Yang; Stephen Eckert; Sandra Rojas-Caro; Anthony G Quinn
Journal:  N Engl J Med       Date:  2015-09-10       Impact factor: 91.245

9.  Correction to: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies.

Authors:  Suresh Vijay; Anais Brassier; Arunabha Ghosh; Simona Fecarotta; Florian Abel; Sachin Marulkar; Simon A Jones
Journal:  Orphanet J Rare Dis       Date:  2021-03-01       Impact factor: 4.123

10.  Sebelipase alfa for lysosomal acid lipase deficiency: 5-year treatment experience from a phase 2 open-label extension study.

Authors:  Vĕra Malinová; Manisha Balwani; Reena Sharma; Jean-Baptiste Arnoux; John Kane; Chester B Whitley; Sachin Marulkar; Florian Abel
Journal:  Liver Int       Date:  2020-08-09       Impact factor: 5.828
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.