Personalized and targeted medicine for rare lung and other cancers

Proteomic signatures improve risk prediction for common and rare diseases  Nature.com

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets. Identifying

The post Looking for Opportunities to Accelerate Clinical Research in Rare Diseases appeared first on LifeSciVC.

US Food and Drug Administration has granted breakthrough therapy designation for two experimental drugs for rare diseases conducted by the biotechnology company Shire.

They seek urgent intervention by government, courts, and lawmakers into the issue  

Monopoly could hinder Union Health Ministry’s efforts “to benefit from domestic production and competitive pricing through pooled procurement strategies”

Treatment for six rare disease patients in Karnataka, under the Centre’s grant of ₹50 lakh each, was stopped recently because the funding limit had been reached. Afshan Yasmeen The Hindu speaks to patients and families and takes an overview of the treatment scenario

Rare diseases in childhood can be debilitating and require lifelong care. Since 1983, the Orphan Drug Act incentives have stimulated the development and significantly improved the availability of treatment products for patients with rare diseases.

We report an increasing pediatric orphan product designations and approvals from 2000 to 2009. The trend indicates that the Orphan Drug Act has continued to address this important unmet need. (Read the full article)

In this essay, a critical care pediatric hospitalist finds herself on the other side of the office table advocating for the specific medical care needed to address her son’s rare skeletal dysplasia and her search for a pediatric specialist with whom to travel on this quest.