NPD

National Niemann-Pick Disease Foundation

Publications

All publications
Fda Advisory Committee Votes Favorably That the Data Support Arimoclomol as Effective Treatment for Patients With Niemann-Pick Disease Type C

Fda Advisory Committee Votes Favorably That the Data Support Arimoclomol as...

2024

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Rare Disease Week on Capitol Hill

Rare Disease Week on Capitol Hill

2024

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Community Update

Community Update

2024

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Rare Disease Week on Capitol Hill

Rare Disease Week on Capitol Hill

2024

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Nord_signonrecs_hb616_sb2156_rdac_ms

Nord_signonrecs_hb616_sb2156_rdac_ms

2024

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Rare Disease Week on Capitol Hill

Rare Disease Week on Capitol Hill

2024

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Living With a Rare or Genetic Condition?

Living With a Rare or Genetic Condition?

2024

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N -Acetyl- -Leucine and Neurodegenerative Disease

N -Acetyl- -Leucine and Neurodegenerative Disease

2024

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Zevra Therapeutics’ Miplyffa™ (Arimoclomol) Receives u.s. Fda Approval as Treatment for Niemann-Pick Disease Type C

Zevra Therapeutics’ Miplyffa™ (Arimoclomol) Receives u.s. Fda Approval as...

2024

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Zevra Therapeutics Receives Fda Acceptance of Resubmission of Nda for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

Zevra Therapeutics Receives Fda Acceptance of Resubmission of Nda for...

2024

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