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1-7 of 7 results for ultra-orphan disease

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Free access

Review article

First published 2017-3

Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician’s guide

Morag Griffin, Talha Munir

Therapeutic Advances in Hematology

Open access

Research article

First published 2025-3

Ultra-Orphan drug development for GNE Myopathy: A synthetic literature review and meta-analysis

Naoki Suzuki, Madoka Mori-Yoshimura, Ichizo Nishino [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2019

Evaluation of quantitative signal detection in EudraVigilance for orphan drugs: possible risk of false negatives

Marco Sardella, Calin Lungu

Therapeutic Advances in Drug Safety

Retricted access

Research article

First published 2017-06

Investor Outlook: The Unanswered Questions

Joshua Schimmer, Steven Breazzano

Human Gene Therapy Clinical Development

Open access

Research article

First published 2018-12-20

Conducting an investigator-initiated randomized double-blinded intervention trial in acute decompensation of inborn errors of metabolism: Lessons from the N-Carbamylglutamate Consortium

Nicholas Ah Mew, Avital Cnaan, Robert McCarter [...]

Translational Science of Rare Diseases

Open access

Review article

First published 2021

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

Yoshitsugu Aoki, Matthew J.A. Wood

Journal of Neuromuscular Diseases

Open access

Review article

First published 2020

A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?

Morag Griffin, Richard Kelly, Alexandra Pike

Therapeutic Advances in Rare Disease