FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation

Case Medical Research ◽

10.31525/cmr-2416295 ◽

2019 ◽

Author(s):

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Targeted Treatment ◽

Accelerated Approval

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FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation

Case Medical Research ◽

10.31525/cmr-24163ba ◽

2019 ◽

Author(s):

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Targeted Treatment ◽

Accelerated Approval

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FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

Case Medical Research ◽

10.31525/cmr-29dc5f3 ◽

2016 ◽

Author(s):

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Accelerated Approval

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Impact of Bisphosphonates on Survival for Patients With Duchenne Muscular Dystrophy

Yearbook of Neurology and Neurosurgery ◽

10.1016/j.yneu.2011.04.066 ◽

2011 ◽

Vol 2011 ◽

pp. 198-199

Author(s):

G.N. Breningstall

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy

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Treatment of Duchenne muscular dystrophy with cyclosporin A – A randomized, double-blind, placebo controlled trial

Neuropediatrics ◽

10.1055/s-0029-1215757 ◽

2008 ◽

Vol 39 (05) ◽

Author(s):

J Kirschner ◽

J Schessl ◽

G Ihorst ◽

R Korinthenberg

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Cyclosporin A ◽

Controlled Trial ◽

Double Blind ◽

Double Blind Placebo

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Health- related quality of life in patients with Duchenne Muscular Dystrophy

Neuropediatrics ◽

10.1055/s-0031-1273950 ◽

2011 ◽

Vol 42 (S 01) ◽

Author(s):

B Geers ◽

S Elsenbruch ◽

J Schmid ◽

U Schara

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Health Related Quality ◽

Related Quality ◽

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Integrating pediatric palliative care across the spectrum of life threatening pediatric neuromuscular disorders focused on Spinal muscular atrophy type I and Duchenne muscular dystrophy

Neuropediatrics ◽

10.1055/s-0031-1274094 ◽

2011 ◽

Vol 42 (S 01) ◽

Author(s):

M von der Hagen ◽

M Smitka ◽

A Pyper ◽

M Breiting ◽

A Müller ◽

...

Keyword(s):

Palliative Care ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Neuromuscular Disorders ◽

Pediatric Palliative Care ◽

Type I ◽

Spinal Muscular Atrophy Type ◽

Life Threatening

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Growth and psychomotor development of patients with Duchenne Muscular Dystrophy

Neuropediatrics ◽

10.1055/s-0032-1307138 ◽

2012 ◽

Vol 43 (02) ◽

Author(s):

E Sarrazin ◽

M von der Hagen ◽

U Schara ◽

K von Au ◽

A Kaindl

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Psychomotor Development

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Current care practice in Duchenne muscular dystrophy in Europe - results of the CARE-NMD cross-sectional survey

Neuropediatrics ◽

10.1055/s-0033-1337747 ◽

2013 ◽

Vol 44 (02) ◽

Author(s):

J Vry ◽

K Gramsch ◽

K Bushby ◽

H Lochmüller ◽

S Rodger ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Care Practice ◽

Cross Sectional Survey ◽

Cross Sectional

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Use of Complementary and Alternative Medicine (CAM) in Patients with X-Linked Recessive Duchenne Muscular Dystrophy (DMD)

Neuropediatrics ◽

10.1055/s-0036-1583655 ◽

2016 ◽

Vol 47 (S 01) ◽

Author(s):

E. Giagkou ◽

T. Zuzak ◽

U. Schara

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Alternative Medicine ◽

Complementary And Alternative Medicine ◽

Complementary And Alternative

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The Six-Minute Walk Test (6MWT) as Endpoint in Duchenne Muscular Dystrophy (DMD) Clinical Studies

Neuropediatrics ◽

10.1055/s-0036-1583649 ◽

2016 ◽

Vol 47 (S 01) ◽

Author(s):

S.-M. Park ◽

C. McDonald ◽

H. Sweeney ◽

X. Luo ◽

G. Elfring ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Clinical Studies ◽

Walk Test ◽

Six Minute Walk Test ◽

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