Search the collection

Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 21
ArticlesPeer ReviewedFull Text
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Author / Creator:Mercuri, Eugenio ; Vilchez, Juan J ; Boespflug-Tanguy, Odile ; Zaidman, Craig M ; Mah, Jean K ; Goemans, Nathalie ; Müller-Felber, Wolfgang ; Niks, Erik H ; Schara-Schmidt, Ulrike ; Bertini, Enrico ; Comi, Giacomo P ; Mathews, Katherine D ; Servais, Laurent ; Vandenborne, Krista ; Johannsen, Jessika ; Messina, Sonia ; Spinty, Stefan ; McAdam, Laura ; Selby, Kathryn ; Byrne, Barry ; Laverty, Chamindra G ; Carroll, Kevin ; Zardi, Giulia ; Cazzaniga, Sara ; Coceani, Nicoletta ; Bettica, Paolo ; McDonald, Craig M ; Acsadi, Gyula ; Baranello, Giovanni ; Blaschek, Astrid ; Brandsema, John ; Brogna, Claudia ; Bruno, Claudio ; Connolly, Anne ; de Groot, Imelda ; De Waele, Liesbeth ; Finanger, Erika ; Finkel, Richard ; Gidaro, Teresa ; Guglieri, Michaela ; Harper, Amy ; Lopez Lobato, Mercedes ; Madruga Garrido, Marcos ; Magri, Francesca ; Manousakis, Georgios ; Masson, Riccardo ; Monduy, Migvis ; Muelas Gomez, Nuria ; Munell, Francina ; Nascimento, Andres ; Nevo, Yoram ; Pereon, Yann ; Phan, Han ; Sansone, Valeria ; Scoto, Mariacristina ; Vucinic, Dragana ; Willis, Tracey
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2024-04, Vol.23 (4), p.393-403
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 22
ArticlesPeer ReviewedFull Text
In Vivo NADH Fluorescence Monitoring as an Assay for Cellular Damage in Photodynamic Therapy
Author / Creator:Pogue, Brian W. ; Pitts, Jonathan D. ; Mycek, Mary-Ann ; Sloboda, Roger D. ; Wilmot, Carmen M. ; Brandsema, John F. ; O'Hara, Julia A.
Publisher:Oxford, UK: Blackwell Publishing Ltd
Journal title:Photochemistry and photobiology, 2001-12, Vol.74 (6), p.817-824
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 23
ArticlesPeer ReviewedFull Text
Novel approaches to analysis of the North Star Ambulatory Assessment : Observations from a phase 2 trial
Author / Creator:Muntoni, Francesco ; Guglieri, Michela ; Mah, Jean K ; Wagner, Kathryn R ; Brandsema, John F ; Butterfield, Russell J ; McDonald, Craig M ; Mayhew, Anna G ; Palmer, Jeffrey P ; Marraffino, Shannon ; Charnas, Lawrence ; Mercuri, Eugenio
Publisher:Public Library of Science
Journal title:PloS one, 2022-08, Vol.17 (8), p.e0272858
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 24
ArticlesPeer ReviewedFull Text
Novel approaches to analysis of the North Star Ambulatory Assessment
Author / Creator:Muntoni, Francesco ; Guglieri, Michela ; Mah, Jean K ; Wagner, Kathryn R ; Brandsema, John F ; Butterfield, Russell J ; McDonald, Craig M ; Mayhew, Anna G ; Palmer, Jeffrey P ; Marraffino, Shannon ; Charnas, Lawrence ; Mercuri, Eugenio
Publisher:Public Library of Science
Journal title:PloS one, 2022-08, Vol.17 (8), p.e0272858
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 25
ArticlesFull Text
Clinically Relevant Challenges and Roadblocks of Gene Therapies for Muscular Dystrophies: John Brandsema, MD
Author / Creator:Brandsema, John F
Publisher:Cranbury: Intellisphere, LLC
Journal title:Neurology live (Print), 2025-03
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 26
ArticlesFull Text
Navigating the Future of Gene Therapy in Neuromuscular Disorders: John Brandsema, MD
Author / Creator:Brandsema, John F
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2025-02
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 27
ArticlesFull Text
Genetic Neurodegenerative Disorders: Close to a Symptom-Free Life?
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2022-02
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 28
ArticlesFull Text
Role of Combination Therapies in SMA Management
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-07
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 29
ArticlesFull Text
Using Small Molecule Treatment for SMA Management
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-07
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 30
ArticlesFull Text
SMA: Patient Adherence to Therapy
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-07
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 31
ArticlesFull Text
Role of Nusinersen in SMA: Efficacy and Treatment Response
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-07
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 32
ArticlesFull Text
Role of Gene Transfer Therapy in SMA
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-07
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 33
ArticlesFull Text
Targeted Therapies for SMA Management: Nusinersen
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 34
ArticlesFull Text
Early Referral to Neurologists for SMA Diagnosis
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 35
ArticlesFull Text
Educating Primary Care Physicians on the Diagnosis of SMA
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 36
ArticlesFull Text
Early Detection and Screening of Spinal Muscular Atrophy
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 37
ArticlesFull Text
Managing SMA in Clinical Practice
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 38
ArticlesFull Text
Classification of Spinal Muscular Atrophy (SMA)
Author / Creator:Brandsema, John
Publisher:Cranbury: MultiMedia Healthcare Inc
Journal title:Neurology live (Print), 2021-06
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 39
ArticlesFull Text
Key Considerations in Treatment of Spinal Muscular Atrophy / Considerações essenciais para o tratamento da atrofia muscular espinhal
Author / Creator:Brandsema, John F
Publisher:Cranbury: Intellisphere, LLC
Journal title:Neurology live (Print), 2023-03
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.
Export this record
Export to excel
Print
Export record to excel
File type to download
Beta version
Select record 40
ArticlesFull Text
Rare Diseases Awareness and Educational Needs / Conscientização sobre doenças raras e necessidades educacionais
Author / Creator:Brandsema, John F
Publisher:Cranbury: Intellisphere, LLC
Journal title:Neurology live (Print), 2023-03
View online as a Library member
Access ebook if you are in the Library. Registered NSW residents may log in from home.

Showing 21 to 40 of 45 results

Export record to excel

Export this record

Export record to excel

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Export this record

Export record to excel

In Vivo NADH Fluorescence Monitoring as an Assay for Cellular Damage in Photodynamic Therapy

Export this record

Export record to excel

Novel approaches to analysis of the North Star Ambulatory Assessment : Observations from a phase 2 trial

Export this record

Export record to excel

Novel approaches to analysis of the North Star Ambulatory Assessment

Export this record

Export record to excel

Clinically Relevant Challenges and Roadblocks of Gene Therapies for Muscular Dystrophies: John Brandsema, MD

Export this record

Export record to excel

Navigating the Future of Gene Therapy in Neuromuscular Disorders: John Brandsema, MD

Export this record

Export record to excel

Genetic Neurodegenerative Disorders: Close to a Symptom-Free Life?

Export this record

Export record to excel

Role of Combination Therapies in SMA Management

Export this record

Export record to excel

Using Small Molecule Treatment for SMA Management

Export this record

Export record to excel

SMA: Patient Adherence to Therapy

Export this record

Export record to excel

Role of Nusinersen in SMA: Efficacy and Treatment Response

Export this record

Export record to excel

Role of Gene Transfer Therapy in SMA

Export this record

Export record to excel

Targeted Therapies for SMA Management: Nusinersen

Export this record

Export record to excel

Early Referral to Neurologists for SMA Diagnosis

Export this record

Export record to excel

Educating Primary Care Physicians on the Diagnosis of SMA

Export this record

Export record to excel

Early Detection and Screening of Spinal Muscular Atrophy

Export this record

Export record to excel

Managing SMA in Clinical Practice

Export this record

Export record to excel

Classification of Spinal Muscular Atrophy (SMA)

Export this record

Export record to excel

Key Considerations in Treatment of Spinal Muscular Atrophy / Considerações essenciais para o tratamento da atrofia muscular espinhal

Export this record

Export record to excel

Rare Diseases Awareness and Educational Needs / Conscientização sobre doenças raras e necessidades educacionais

Refine your search