A call to arms against ultra-rare diseases

A call to arms against ultra-rare diseases

The not-for profit foundation n-Lorem seeks to provide experimental antisense oligonucleotide treatments to patients with ultra-rare disease free for their entire lifetime.

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Bibliographische Detailangaben
Veröffentlicht in:Nature biotechnology 2021-06, Vol.39 (6), p.671-677
1. Verfasser: Crooke, Stanley T.
Format: Artikel
Sprache:eng
Schlagworte:
692/699
706/648
Agriculture
Aims and objectives
Antisense oligonucleotides
Bioinformatics
Biomedical and Life Sciences
Biomedical Engineering/Biotechnology
Biomedicine
Biotechnology
Comment
Disease
Finance
Foundations (Endowments)
Good Manufacturing Practice
Life Sciences
Medical research
Medicine, Experimental
Mutation
Patients
Rare diseases
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Beschreibung
Zusammenfassung:The not-for profit foundation n-Lorem seeks to provide experimental antisense oligonucleotide treatments to patients with ultra-rare disease free for their entire lifetime.
ISSN:1087-0156
1546-1696
DOI:10.1038/s41587-021-00945-0