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Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions

Drugs

Nov 18, 2019

Stéphane AuvinJ Helen Cross

Abstract

Rare diseases provide a challenge in the evaluation of new therapies. However, orphan drug development is of increasing interest because of the legislation enabling facilitated support by regulatory agencies through scientific advice, and the protection of the molecules with orphan desi...read more

Mentioned in this Paper

Drugs, Orphan
Infantile Severe Myoclonic Epilepsy
Epilepsy
Gene Mutation
Evaluation
Pharmacologic Substance
Lennox-Gastaut syndrome
Clinical Research
Compound (Substance)
Infantile Spasms
8
Paper Details
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Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions

Drugs

Nov 18, 2019

Stéphane AuvinJ Helen Cross

PMID: 31734883

DOI: 10.1007/s40265-019-01223-9

Abstract

Rare diseases provide a challenge in the evaluation of new therapies. However, orphan drug development is of increasing interest because of the legislation enabling facilitated support by regulatory agencies through scientific advice, and the protection of the molecules with orphan desi...read more

Mentioned in this Paper

Drugs, Orphan
Infantile Severe Myoclonic Epilepsy
Epilepsy
Gene Mutation
Evaluation
Pharmacologic Substance
Lennox-Gastaut syndrome
Clinical Research
Compound (Substance)
Infantile Spasms
8
Paper Details
References
  • References
  • Citations
  • finger pointing at paper

    References currently unavailable

    We're still populating references for this paper, please check back later.
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