(Q40122857)

17 September 2017

Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design (English)

1 reference

17 September 2017

P Kaufmann

1 reference

17 September 2017

F Muntoni

series ordinal

1 reference

17 September 2017

International Coordinating Committee for SMA Subcommittee on SMA Clinical Trial Design

series ordinal

1 reference

17 September 2017

14 February 2007

1 reference

17 September 2017

Neuromuscular Disorders

1 reference

17 September 2017

1 reference

17 September 2017

1 reference

17 September 2017

499-505

1 reference

Neuroprotective agents for clinical trials in ALS: a systematic assessment

17 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Dichloroacetate causes toxic neuropathy in MELAS: a randomized, controlled clinical trial

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Challenges and opportunities in clinical trials for spinal muscular atrophy

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Natural history of denervation in SMA: relation to age, SMN2 copy number, and function

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Concerns about the design of clinical trials for spinal muscular atrophy

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

A confirmatory dose-ranging study of riluzole in ALS. ALS/Riluzole Study Group-II.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Optimal two-stage designs for phase II clinical trials

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

A two-stage design for a phase II clinical trial of coenzyme Q10 in ALS.

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

20 August 2018

The utility of futility.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Sequential designs for clinical trials in amyotrophic lateral sclerosis

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Sleep disordered breathing in spinal muscular atrophy

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

A randomized sequential trial of creatine in amyotrophic lateral sclerosis

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Outcome measures for pediatric spinal muscular atrophy.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

On the use of nonparametric curves in phase I trials with low toxicity tolerance.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Motor unit number estimation in infants and children with spinal muscular atrophy

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Chaos in the classification of SMA: a possible resolution.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Reliability of the Hammersmith functional motor scale for spinal muscular atrophy in a multicentric study

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3250230

Sequential designs for phase I clinical trials with late-onset toxicities

2 December 2018

1 reference

12 December 2020

A simple technique to evaluate model sensitivity in the continual reassessment method

1 reference

12 December 2020

A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group

1 reference

12 December 2020

An optimal three-stage design for phase II clinical trials

1 reference

12 December 2020

Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II

1 reference

12 December 2020

A motor function measure for neuromuscular diseases. Construction and validation study

1 reference

12 December 2020

[Spinal muscular atrophy. A 4-year prospective, multicenter, longitudinal study (168 cases)]

1 reference

12 December 2020

[A motor function measurement scale for neuromuscular diseases - description and validation study]

1 reference

12 December 2020

The gross motor function measure™ is a valid and sensitive outcome measure for spinal muscular atrophy

1 reference

12 December 2020

10.1016/J.NMD.2006.12.001

Identifiers

DOI

1 reference

17 September 2017

1 reference

17 September 2017

PubMed publication ID

17300938

1 reference