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Pat Furlong Selected Research

Therapeutics

12/2022Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
1/2016Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
2/2015Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints.

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Pat Furlong Research Topics

Disease

6Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker)
12/2022 - 03/2011
3Rare Diseases (Rare Disease)
07/2021 - 02/2015
1Hypersensitivity (Allergy)
12/2022
1Muscular Dystrophies (Muscular Dystrophy)
06/2015

Drug/Important Bio-Agent (IBA)

2Pharmaceutical PreparationsIBA
06/2015 - 02/2015
2Biomarkers (Surrogate Marker)IBA
06/2015 - 02/2015
1chenodeoxycholate sulfate conjugate (CDCS)IBA
12/2022
1Coenzyme A (CoA)IBA
07/2021
1Tadalafil (Cialis)FDA Link
10/2017
1Proteins (Proteins, Gene)FDA Link
06/2015
1Blood Proteins (Serum Proteins)IBA
06/2015
1DNA (Deoxyribonucleic Acid)IBA
03/2011

Therapy/Procedure

3Therapeutics
12/2022 - 02/2015
1Cell- and Tissue-Based Therapy (Cell Therapy)
12/2022
1Intravenous Infusions
12/2022