(Q36375543)

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Unintended effects of orphan product designation for rare neurological diseases.

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

Unintended effects of orphan product designation for rare neurological diseases (English)

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

1 reference

ChEBI release 2020-05-28

1 reference

based on heuristic

inferred from title

1

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

author name string

Araya Puwanant

2

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

Robert C Griggs

3

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

Consortium for Clinical Investigations of Neurological Channelopathies (CINCH) and Inherited Neuropathies Consortium (INC) Consortia of the Rare Disease Clinical Research Network

4

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

publication date

1 October 2012

1 reference

Europe PubMed Central

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16 February 2020

Annals of Neurology

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

72

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

4

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

481-490

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

Pharmacogenomic technologies: a necessary "luxury" for better global public health?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

A comparative study of European rare disease and orphan drug markets

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years

1 reference

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28 June 2018

A randomized study of alglucosidase alfa in late-onset Pompe's disease

1 reference

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28 June 2018

Incentives for drug development--the curious case of colchicine

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Balancing innovation, access, and profits--market exclusivity for biologics

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Pivotal studies of orphan drugs approved for neurological diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Incentives for orphan drug research and development in the United States

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Clinical research for rare disease: opportunities, challenges, and solutions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Prescription drug cost sharing: associations with medication and medical utilization and spending and health.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

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=3490440

28 June 2018

A journey of hope: lessons learned from studies on rare diseases and orphan drugs

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Orphan drug policies: implications for the United States, Canada, and developing countries

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Orphan drug development is progressing too slowly

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Commissioning for rare diseases: view from the frontline

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Orphan drugs and the NHS: should we value rarity?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Two decades of orphan product development

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

High-dose corticotropin (ACTH) versus prednisone for infantile spasms: a prospective, randomized, blinded study.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

Orphan drugs: the question of products liability.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

28 June 2018

A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Using Market-Exclusivity Incentives to Promote Pharmaceutical Innovation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Enzyme replacement for infantile Pompe disease: the first step toward a cure.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Tetrabenazine as antichorea therapy in Huntington disease: a randomized controlled trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

Effect of tetrabenazine on extrapyramidal movement disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3490440

21 September 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Setting Cost-Effectiveness Thresholds As A Means To Achieve Appropriate Drug Prices In Rich And Poor Countries

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Market exclusivity for biologics

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

New drugs and the Kefauver-Harris amendment

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Cyp3A4, Cyp3A5, and MDR-1 genetic influences on tacrolimus pharmacokinetics in renal transplant recipients

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Colchicine's other indication--effect of FDA action

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Orphan diseases: which ones do we adopt?

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Tier 4 drugs and the fraying of the social compact

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Orphan drug pricing may warrant a competition law investigation

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

On rare and "super-rare" diseases: an insight from the Republic of Macedonia

1 reference

https://pubmed.ncbi.nlm.nih.gov/23109143

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1002/ANA.23672

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23109143%20AND%20SRC:MED&resulttype=core&format=json

16 February 2020

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