(Q36945774)

English

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes

scientific article published on June 2013

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scholarly article

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes (English)

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

phase I clinical trial

0 references

0 references

phase II clinical trial

0 references

respiratory failure

1 reference

based on heuristic

inferred from title

author name string

Barbara K Smith

1

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Shelley W Collins

2

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Thomas J Conlon

3

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Cathryn S Mah

4

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Lee Ann Lawson

5

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Anatole D Martin

6

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

David D Fuller

7

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Brian D Cleaver

8

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Nathalie Clément

9

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Dawn Phillips

10

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Saleem Islam

11

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Nicole Dobjia

12

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Barry J Byrne

13

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

publication date

1 June 2013

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Human Gene Therapy

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

24

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

6

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

630-640

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Inspiratory muscle strength training in infants with congenital heart disease and prolonged mechanical ventilation: a case report

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Hypoglossal neuropathology and respiratory activity in pompe mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Inspiratory muscle strength training improves weaning outcome in failure to wean patients: a randomized trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Severe scoliosis in neurodevelopmental disabilities: clinical signs and therapeutic proposals

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Diaphragm motor unit recruitment in rats.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Inspiratory muscle training in a child with nemaline myopathy and organ transplantation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Autophagy in skeletal muscle: implications for Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Neural deficits contribute to respiratory insufficiency in Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Pompe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Measurement of maximal inspiratory pressure in ventilated children

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Rapid disuse atrophy of diaphragm fibers in mechanically ventilated humans.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Clinical approach to the weak patient in the intensive care unit.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Respiratory muscle evaluation of the patient with neuromuscular disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Lung function in white children aged 4 to 19 years: I--Spirometry

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Physiological effects of diaphragm muscle denervation and disuse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Validation of a technique to assess maximal inspiratory pressure in poorly cooperative patients

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

29 September 2017

Diaphragm motor unit recruitment during ventilatory and nonventilatory behaviors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

27 June 2018

Needle electromyography of the diaphragm

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

27 June 2018

Merry christmas for patients with hemophilia B.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

CRIM-negative infantile Pompe disease: 42-month treatment outcome.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Acute progression of neuromuscular findings in infantile Pompe disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Gene transfer into skeletal muscle using novel AAV serotypes.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

POMPE'S DISEASE (DIFFUSE GLYCOGENOSIS) WITH NEURONAL STORAGE

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Determination of maximal voluntary ventilation in children with cystic fibrosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Tracheobronchomalacia and tracheal hemorrhage in patients with Duchenne muscular dystrophy receiving long-term ventilation with uncuffed tracheostomies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Forced expiratory parameters in healthy preschool children (3-6 years of age).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Maximal static respiratory pressures in children and adolescents.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Inspiratory muscle training in patients with chronic obstructive pulmonary disease: structural adaptation and physiologic outcomes.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Effect of inspiratory muscle strength training on inspiratory motor drive and RREP early peak components.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

ATS/ERS Statement on respiratory muscle testing.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Electrophysiological monitoring in neurological respiratory insufficiency

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Nervous system in Pompe's disease. Ultrastructure and biochemistry

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3689178

4 September 2018

Rapidly progressive diaphragmatic weakness and injury during mechanical ventilation in humans

1 reference

https://pubmed.ncbi.nlm.nih.gov/23570273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Pulmonary complications of neuromuscular disease: a respiratory mechanics perspective

1 reference

https://pubmed.ncbi.nlm.nih.gov/23570273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Respiratory muscle testing: a valuable tool for children with neuromuscular disorders

1 reference

https://pubmed.ncbi.nlm.nih.gov/23570273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1089/HUM.2012.250

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

1 reference

Europe PubMed Central

PMC publication ID

17 August 2017

ResearchGate publication ID

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