Accueil
Fatbardha Varfaj
Fatbardha Varfaj
Affiliations
Astellas Gene Therapies, San Francisco, CA, USA.
Publications (4)
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
The Lancet. Neurology
Avec:
Perry B Shieh
,
Nancy L Kuntz
,
James J Dowling
,
Wolfgang Müller-Felber
,
Carsten G Bönnemann
,
Andreea M Seferian
,
Laurent Servais
,
Barbara K Smith
,
Francesco Muntoni
,
Astrid Blaschek
,
A Reghan Foley
,
Dimah N Saade
,
Sarah Neuhaus
,
Lindsay N Alfano
,
Alan H Beggs
,
Ana Buj-Bello
,
Martin K Childers
,
Tina Duong
,
Robert J Graham
,
Minal Jain
,
Julie Coats
,
Vicky MacBean
,
Emma S James
,
Jun Lee
,
Fulvio Mavilio
,
Weston Miller
,
Fatbardha Varfaj
,
Michael Murtagh
,
Cong Han
,
Mojtaba Noursalehi
,
Michael W Lawlor
,
Suyash Prasad
,
Salvador Rico
Acid α-glucosidase (GAA) activity and glycogen content in muscle biopsy specimens of patients with Pompe disease: A systematic review.
Molecular genetics and metabolism reports
Avec:
Benedikt Schoser
,
Nina Raben
,
Fatbardha Varfaj
,
Mark Walzer
,
Antonio Toscano
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy.
American journal of human genetics
Avec:
Gaia Andreoletti
,
Oriana Romano
,
Hsin-Jung Chou
,
Mahjoubeh J Sefid-Dashti
,
Andrea Grilli
,
Clarice Chen
,
Neema Lakshman
,
Pravin Purushothaman
,
Fatbardha Varfaj
,
Fulvio Mavilio
,
Silvio Bicciato
,
Fabrizia Urbinati
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
EBioMedicine
Avec:
Michael W Lawlor
,
Benedikt Schoser
,
Marta Margeta
,
Caroline A Sewry
,
Karra A Jones
,
Perry B Shieh
,
Nancy L Kuntz
,
Barbara K Smith
,
James J Dowling
,
Wolfgang Müller-Felber
,
Carsten G Bönnemann
,
Andreea M Seferian
,
Astrid Blaschek
,
Sarah Neuhaus
,
A Reghan Foley
,
Dimah N Saade
,
Etsuko Tsuchiya
,
Ummulwara R Qasim
,
Margaret Beatka
,
Mariah J Prom
,
Emily Ott
,
Susan Danielson
,
Paul Krakau
,
Suresh N Kumar
,
Hui Meng
,
Mark Vanden Avond
,
Clive Wells
,
Heather Gordish-Dressman
,
Alan H Beggs
,
Sarah Christensen
,
Edward Conner
,
Emma S James
,
Jun Lee
,
Chanchal Sadhu
,
Weston Miller
,
Bryan Sepulveda
,
Fatbardha Varfaj
,
Suyash Prasad
,
Salvador Rico
Réseau de co-auteurs
Perry B Shieh
2 collaborations
Nancy L Kuntz
2 collaborations
James J Dowling
2 collaborations
Wolfgang Müller-Felber
2 collaborations
Carsten G Bönnemann
2 collaborations
Andreea M Seferian
2 collaborations
Barbara K Smith
2 collaborations
Astrid Blaschek
2 collaborations
A Reghan Foley
2 collaborations
Dimah N Saade
2 collaborations
Sarah Neuhaus
2 collaborations
Alan H Beggs
2 collaborations
Emma S James
2 collaborations
Jun Lee
2 collaborations
Fulvio Mavilio
2 collaborations
Weston Miller
2 collaborations
Michael W Lawlor
2 collaborations
Suyash Prasad
2 collaborations
Salvador Rico
2 collaborations
Benedikt Schoser
2 collaborations
Laurent Servais
1 collaboration
Francesco Muntoni
1 collaboration
Lindsay N Alfano
1 collaboration
Ana Buj-Bello
1 collaboration
Martin K Childers
1 collaboration
Tina Duong
1 collaboration
Robert J Graham
1 collaboration
Minal Jain
1 collaboration
Julie Coats
1 collaboration
Vicky MacBean
1 collaboration
Michael Murtagh
1 collaboration
Cong Han
1 collaboration
Mojtaba Noursalehi
1 collaboration
Nina Raben
1 collaboration
Mark Walzer
1 collaboration
Antonio Toscano
1 collaboration
Gaia Andreoletti
1 collaboration
Oriana Romano
1 collaboration
Hsin-Jung Chou
1 collaboration
Mahjoubeh J Sefid-Dashti
1 collaboration
Andrea Grilli
1 collaboration
Clarice Chen
1 collaboration
Neema Lakshman
1 collaboration
Pravin Purushothaman
1 collaboration
Silvio Bicciato
1 collaboration
Fabrizia Urbinati
1 collaboration
Marta Margeta
1 collaboration
Caroline A Sewry
1 collaboration
Karra A Jones
1 collaboration
Etsuko Tsuchiya
1 collaboration
Ummulwara R Qasim
1 collaboration
Margaret Beatka
1 collaboration
Mariah J Prom
1 collaboration
Emily Ott
1 collaboration
Susan Danielson
1 collaboration
Paul Krakau
1 collaboration
Suresh N Kumar
1 collaboration
Hui Meng
1 collaboration
Mark Vanden Avond
1 collaboration
Clive Wells
1 collaboration
Heather Gordish-Dressman
1 collaboration
Sarah Christensen
1 collaboration
Edward Conner
1 collaboration
Chanchal Sadhu
1 collaboration
Bryan Sepulveda
1 collaboration