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A further case with chronic hemolysis, CIDP and lethal cerebral vasculitis due to a CD59 mutation

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S143-S143

2016

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T.I.3 The rationale for immunosuppressive treatment in DMD

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.830-830

Korinthenberg, R

2008

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Handlungsempfehlung nach der Leitlinie „Guillain-Barré Syndrom im Kindes- und Jugendalter

Monatsschrift Kinderheilkunde, 2013-12, Vol.161 (12), p.1201-1202

Korinthenberg, R.

2013

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Echocardiographic and electrocardiographic findings of cardiomyopathy in Duchenne and Becker-Kiener muscular dystrophies

Pediatric cardiology, 2005-02, Vol.26 (1), p.66-72

2005

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Six minute walk test in type III spinal muscular atrophy: A 12month longitudinal study

Neuromuscular disorders : NMD, 2013-08, Vol.23 (8), p.624-628

2013

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Obesity after childhood craniopharyngioma - German multicenter study on pre-operative risk factors and quality of life

Klinische Pädiatrie, 2001-07, Vol.213 (4), p.244-249

2001

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The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S271-S271

2015

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Musculoskeletal pain: a new algorithm for differential diagnosis of a cardinal symptom in pediatrics

Klinische Padiatrie, 2014-04, Vol.226 (2), p.86

2014

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Prospective study on anti-ganglioside antibodies in childhood Guillain–Barré syndrome

Archives of disease in childhood, 2007-01, Vol.92 (1), p.48-52

2007

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OP64 – 2760: Oligoclonal bands predict multiple sclerosis in children with isolated optic neuritis

European journal of paediatric neurology, 2015-05, Vol.19, p.S20-S21

2015

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P.188 - A further case with chronic hemolysis, CIDP and lethal cerebral vasculitis due to a CD59 mutation

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S143-S143

2016

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Clinical and Experimental Results on Cardiac Troponin Expression in Duchenne Muscular Dystrophy

Clinical chemistry (Baltimore, Md.), 2001-03, Vol.47 (3), p.451-458

2001

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G.P.2 Mutations in the human isoprenoid synthase domain containing gene are a common cause of congenital and limb girdle muscular dystrophies

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.812-812

2012

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G.P.3.09 Mutation in PDHA1 gene mimicking a Guillain-Barré-Syndrome

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.754-754

2008

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M.P.1.09 TREAT-NMD Clinical Trials Coordination Centre: Efficiency of networking

Neuromuscular disorders : NMD, 2009-09, Vol.19 (8), p.548-549

2009

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Natural history and treatment effects in Guillain-Barré syndrome: a multicentre study

Archives of disease in childhood, 1996-04, Vol.74 (4), p.281-287

1996

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Compound heterozygosity of the protein S-gene as a cause of severe cerebral sinovenous thrombosis in a 7-year-old child

Klinische Padiatrie, 2010-05, Vol.222 (3), p.194

2010

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The Heart in Friedreich Ataxia: Definition of Cardiomyopathy, Disease Severity, and Correlation With Neurological Symptoms

Circulation (New York, N.Y.), 2012-04, Vol.125 (13), p.1626-1634

2012

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Identification of seven novel mutations in the GAN gene

Human mutation, 2003-04, Vol.21 (4), p.446-446

2003

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P3.52 FOR-DMD: double-blind randomized trial to optimize steroid regime in Duchenne Muscular Dystrophy (DMD)

Neuromuscular disorders : NMD, 2010-10, Vol.20 (9), p.657-657

2010

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Aicardi-Goutières-Syndrom: Seltene genetische Erkrankung imitiert intrauterine Virusinfektion

Monatsschrift Kinderheilkunde, 2009-05, Vol.157 (5), p.473-478

2009

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Peripheral Nerve Demyelination Caused by a Mutant Rho GTPase Guanine Nucleotide Exchange Factor, Frabin/FGD4

American journal of human genetics, 2007-07, Vol.81 (1), p.158-164

2007

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T.P.3.08 The TREAT-NMD Clinical Trials Coordination Centre (CTCC)

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.796-796

2008

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Expansion of the genotypic and phenotypic spectrum in patients with KRAS germline mutations

Journal of medical genetics, 2007-02, Vol.44 (2), p.131-135

2007

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Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

Orphanet journal of rare diseases, 2015-05, Vol.10 (1), p.65-65, Article 65

2015

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A further case with chronic hemolysis, CIDP and lethal cerebral vasculitis due to a CD59 mutation

T.I.3 The rationale for immunosuppressive treatment in DMD

Handlungsempfehlung nach der Leitlinie „Guillain-Barré Syndrom im Kindes- und Jugendalter

Echocardiographic and electrocardiographic findings of cardiomyopathy in Duchenne and Becker-Kiener muscular dystrophies

Six minute walk test in type III spinal muscular atrophy: A 12month longitudinal study

Obesity after childhood craniopharyngioma - German multicenter study on pre-operative risk factors and quality of life

The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

Musculoskeletal pain: a new algorithm for differential diagnosis of a cardinal symptom in pediatrics

Prospective study on anti-ganglioside antibodies in childhood Guillain–Barré syndrome

OP64 – 2760: Oligoclonal bands predict multiple sclerosis in children with isolated optic neuritis

P.188 - A further case with chronic hemolysis, CIDP and lethal cerebral vasculitis due to a CD59 mutation

Clinical and Experimental Results on Cardiac Troponin Expression in Duchenne Muscular Dystrophy

G.P.2 Mutations in the human isoprenoid synthase domain containing gene are a common cause of congenital and limb girdle muscular dystrophies

G.P.3.09 Mutation in PDHA1 gene mimicking a Guillain-Barré-Syndrome

M.P.1.09 TREAT-NMD Clinical Trials Coordination Centre: Efficiency of networking

Natural history and treatment effects in Guillain-Barré syndrome: a multicentre study

Compound heterozygosity of the protein S-gene as a cause of severe cerebral sinovenous thrombosis in a 7-year-old child

The Heart in Friedreich Ataxia: Definition of Cardiomyopathy, Disease Severity, and Correlation With Neurological Symptoms

Identification of seven novel mutations in the GAN gene

P3.52 FOR-DMD: double-blind randomized trial to optimize steroid regime in Duchenne Muscular Dystrophy (DMD)

Aicardi-Goutières-Syndrom: Seltene genetische Erkrankung imitiert intrauterine Virusinfektion

Peripheral Nerve Demyelination Caused by a Mutant Rho GTPase Guanine Nucleotide Exchange Factor, Frabin/FGD4

T.P.3.08 The TREAT-NMD Clinical Trials Coordination Centre (CTCC)

Expansion of the genotypic and phenotypic spectrum in patients with KRAS germline mutations

Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

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