Innovations in Therapy Development for Rare Diseases Through the Rare Disease Cures Accelerator-Data and Analytics Platform

DOI: 10.1007/s43441-022-00408-x Published: 2022/9 Keywords: Analysis platform; Data sharing; Rare disease Authors: Jane Larkindale Page Count: 9

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Innovations In Therapy Development For Rare Diseases Through …

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Jun 6, 2022 · Rare diseases impact the lives of an estimated 350 million people worldwide, and yet about 90% of rare diseases remain without an approved treatment. New technologies have …

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Jun 6, 2022 · Rare diseases impact the lives of an estimated 350 million people worldwide, and yet about 90% of rare diseases remain without an approved treatment. New technologies have …

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FAQs about Innovations in Therapy Development for Rare Diseases Through the Rare Disease Cures Accelerator-Data and Analytics Platform Coupon?

Why do we need innovative treatments for rare diseases?

For people living with rare genetic diseases – many of which can be debilitating or life-threatening – the need for innovative treatments is urgent. Only 5% of the 7,000 known rare diseases have an approved treatment, making patients with rare diseases collectively one of the most underserved communities in medicine today. ...

Can gene therapy help people with rare diseases?

The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. ...

Is there a standardised framework for developing a treatment for rare diseases?

New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. ...

What are the proposed actions in rare disease drug development?

Proposed actions include (1) advising on the range of available regulatory pathways and flexibilities and (2) providing clarity on how to comply with regulatory policies, apply guidance, and meet requirements in rare disease drug development. ...

How has a focus on rare diseases changed the world?

In recent decades, a focus on rare diseases has resulted in an increased awareness of the community and specific diseases, which has progressed the field. This can be seen in media visibility and overall attention, including funding of rare disease research and targeted drug development by governments. ...

What is cure rare disease?

Cure Rare Disease is a non-profit biotech focused on the development of gene editing, replacement and ASO approaches for neuromuscular diseases through academic collaborations and embedding translational expertise. ...