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Aceneuramic acid

Alternative Names: aceneuramic acid
Latest Update: 2023-10-01
Latest Update Note: PubMed Publication

Product Description

Aceneuramic acid is an N-acetylneuraminic acid that is keto-neuraminic acid in which one of the hydrogens of the amino group is replaced by an acetyl group. It is a member of N-acetylneuraminic acids, a member of acetamides and a 2-oxo monocarboxylic acid. It derives from a keto-neuraminic acid. It is a conjugate acid of an aceneuramate.

Mechanisms of Action: No Mechanism

Novel Mechanism: No

Modality: Small Molecule

Route of Administration: Oral

FDA Designation: None *

Approval Status: Not Approved

Approved Countries: Saudi Arabia

Approved Indications: None

Known Adverse Events: None

Company: Ultragenyx
Company Location: NOVATO CA 94949
Company CEO: Emil D. Kakkis
Additonal Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Aceneuramic acid

Countries in Clinic: Japan

Active Clinical Trial Count: 1

Highest Development Phases

Phase 3: Distal Myopathies|GNE Myopathy

Trial

Phase

Trial Status

Disease

Primary Completion Date

Probability of Success
NPC-09-1

P3

Completed

GNE Myopathy|Distal Myopathies

2022-03-29

85%
NPC-09-1

P3

Completed

GNE Myopathy|Distal Myopathies

2022-03-29

85%

Recent News Events

Date

Type

Title

10/01/2023

PubMed

 [Efficacy of Aceneuramic Acid for Distal Myopathy with Rimmed Vacuoles].

08/11/2023

PubMed

 Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan.

01/01/2023

PubMed

 Phase II/III Study of Aceneuramic Acid Administration for GNE Myopathy in Japan.

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