Progress in molecular biology and translational science addressing the needs of nano-rare patients

Author:

Crooke Stanley T.

Publisher

Elsevier

Reference36 articles.

1. Meeting the needs of patients with ultrarare diseases;Crooke;Trends Mol Med,2022

2. n-Lorem Foundation Notes Latest in Guidance from FDA on Developing ASO Drug Products for Patients With Nano-Rare Diseases. Guidance Issued by FDA Provides a Supportive Environment to Develop Experimental ASO Medicines for Patients With Nano-Rare Disease;Crooke,2022

3. A case for n-of-1 trials—reply;Kravitz;JAMA Int Med,2019

4. Orphans in the market: the history of orphan drug policy;Mikami;Soc His Med,2017

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

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1. Multifunctional nanocomposites for blood cancer;Multifunctional Nanocomposites for Targeted Drug Delivery in Cancer Therapy;2024

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