Abliva’s NV-354 awarded US orphan drug designation for mitochondrial disease

April 18, 2023

Abliva AB’s NV-354 has been granted orphan drug designation by the FDA for the treatment of mitochondrial disease.

BioWorld Science Endocrine/metabolic FDA Orphan drug

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BioWorld

BioWorld briefs for Jan 23, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for Jan. 23, 2026.

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Neurotrimin unveiled as marker in intellectual disability

BioWorld Science

Neurotrimin (NTM) is a member of the IgLON family, the disruption of which has been tied to emotional learning deficits and anxiety-like behavior in animal...

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Biallelic variants in COX18 identified as cause of Charcot-Marie-Tooth disease

BioWorld Science

Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited...

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Cell/gene therapy sector now sustainable; China competition mounting

BioWorld

There was an upbeat message for cell and gene therapy companies in the 2026 industry update presented as the J.P. Morgan Healthcare Conference opened on Monday,...