Meeting the needs of patients with ultrarare diseases

Author:

Crooke Stanley T.ORCID

Publisher

Elsevier BV

Subject

Molecular Biology,Molecular Medicine

Reference32 articles.

1. The Orphan Drug Act revisited;Thomas;JAMA,2019

2. Orphans in the market: the history of orphan drug policy;Mikami;Soc. Hist. Med.,2019

3. A call to arms against ultra-rare diseases;Crooke;Nat. Biotechnol.,2021

4. Addressing the Needs of patients with ultra-rare mutations one patient at a time: the n-Lorem approach;Crooke;Nucleic Acid Ther.,2021

5. The economics of health care quality and medical errors;Andel;J. Health Care Finance,2012

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