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1 . Academic Journal
Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational studyResearch in context
저자
by
René Günther
;
Claudia Diana Wurster
;
Svenja Brakemeier
;
Alma Osmanovic
;
Olivia Schreiber-Katz
, et al.
소스
The Lancet Regional Health. Europe, Vol 39, Iss , Pp 100862- (2024)
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7 . Academic Journal
Cost of illness in inclusion body myositis: results from a cross-sectional study in Germany
저자
by
Katja C. Senn
;
Simone Thiele
;
Karsten Kummer
;
Maggie C. Walter
;
Klaus H. Nagels
.
소스
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-15 (2023)
Open Access (BioMed Central)
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7 . Academic Journal
Inclusion body myositis—health-related quality of life and care situation during phases of the 'patience journey' in Germany: results from a qualitative study
저자
by
Katja C. Senn
;
Simone Thiele
;
Laura Gumbert
;
Sabine Krause
;
Maggie C. Walter
, et al.
소스
Health and Quality of Life Outcomes, Vol 21, Iss 1, Pp 1-11 (2023)
Open Access (BioMed Central)
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7 . Academic Journal
Correction to: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A
저자
by
Shahram Attarian
;
Peter Young
;
Thomas H. Brannagan
;
David Adams
;
Philip Van Damme
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-2 (2024)
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7 . Academic Journal
Misfolding of fukutin-related protein (FKRP) variants in congenital and limb girdle muscular dystrophies
저자
by
Christopher T. Esapa
;
R. A. Jeffrey McIlhinney
;
Adrian J. Waite
;
Matthew A. Benson
;
Jasmin Mirzayan
, et al.
소스
Frontiers in Molecular Biosciences, Vol 10 (2023)
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7 . Academic Journal
Areas of improvement in the medical care of SMA: evidence from a nationwide patient registry in Germany
저자
by
Berenike Leibrock
;
Erik Landfeldt
;
Justine Hussong
;
Tabea Huelle
;
Hannah Mattheus
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-10 (2023)
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7 . Academic Journal
Time to diagnosis of Duchenne muscular dystrophy in Austria and Germany
저자
by
Miriam Hiebeler
;
Simone Thiele
;
Peter Reilich
;
Günther Bernert
;
Maggie C. Walter
.
소스
Scientific Reports, Vol 13, Iss 1, Pp 1-8 (2023)
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7 . Academic Journal
Water T2 could predict functional decline in patients with dysferlinopathy
저자
by
Ursula Moore
;
Ericky Caldas de Almeida Araújo
;
Harmen Reyngoudt
;
Heather Gordish‐Dressman
;
Fiona E. Smith
, et al.
소스
Journal of Cachexia, Sarcopenia and Muscle, Vol 13, Iss 6, Pp 2888-2897 (2022)
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7 . Academic Journal
Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study
저자
by
Astrid Pechmann
;
Max Behrens
;
Katharina Dörnbrack
;
Adrian Tassoni
;
Franziska Wenzel
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-10 (2022)
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7 . Academic Journal
The health-related quality of life, mental health and mental illnesses of patients with inclusion body myositis (IBM): results of a mixed methods systematic review
저자
by
Katja C. Senn
;
Laura Gumbert
;
Simone Thiele
;
Sabine Krause
;
Maggie C. Walter
, et al.
소스
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-15 (2022)
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