Bibliographic Details
| Title: |
Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases. |
| Authors: |
Gallagher, Thomas |
| Source: |
Human Gene Therapy; Jan2025, Vol. 36 Issue 1/2, p1-6, 6p |
| Subject Terms: |
PATIENTS' attitudes, MEDICAL sciences, OPEN Data Protocol, SEVERE combined immunodeficiency, POOR communities, GAIN-of-function mutations |
| Abstract: |
The article "Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases" discusses the efforts of patient advocate Rufang (Kevin) Huang and various organizations to drive advancements in rare disease research and treatment. The focus is on developing innovative approaches to overcome challenges in research, funding, clinical trials, and patient access to new medicines. The article highlights the need for international cooperation, standardized protocols, and novel funding mechanisms to accelerate the development of treatments for the over 7,000 identified rare diseases. It emphasizes the importance of understanding genotype-phenotype relationships, innovative screening methods, preclinical research strategies, and funding mechanisms to bridge the gap between promising therapies and patient access. [Extracted from the article] |
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| Database: |
Biomedical Index |
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