Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

An analysis of original research contributions toward FDA-approved drugs

(2015) Eric V. Patridge et al.   DRUG DISCOVERY TODAY

Experience With the Priority Review Voucher Program for Drug Development

(2015) Aaron S. Kesselheim et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

21st Century Cures Act wins in US House

(2015)   NATURE BIOTECHNOLOGY

Incentives to Repurpose Existing Drugs for Orphan Indications

(2015) Annette K. Kwok et al.   ACS Medicinal Chemistry Letters

The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

(2014) Kristina Sachs-Barrable et al.   DRUG DEVELOPMENT AND INDUSTRIAL PHARMACY

The discovery of medicines for rare diseases

(2014) David C Swinney et al.   Future Medicinal Chemistry

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery

(2013) Jill Wood et al.   DRUG DISCOVERY TODAY

A generalizable pre-clinical research approach for orphan disease therapy

(2012) Chandree L Beaulieu et al.   Orphanet Journal of Rare Diseases

The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

(2011) Brigitta E Miyamoto et al.   Orphanet Journal of Rare Diseases

Therapies for Inborn Errors of Metabolism: What Has the Orphan Drug Act Delivered?

(2010) S. S. Talele et al.   PEDIATRICS

Sanfilippo syndrome: A mini-review

(2008) M. J. Valstar et al.   JOURNAL OF INHERITED METABOLIC DISEASE