On February 29, 2024, Rare Disease Day will take place worldwide to raise awareness for rare diseases. Only 5% of the approximately 6,000 to 10,000 known rare diseases are currently treatable. The research and development of targeted therapeutic approaches is time-consuming, so that many companies shy away from the financial outlay in view of the low number of patients.

Filming Begins With Tech-Evangelist Guy Kawasaki & Dr. Kevin Peng of The House Institute Foundation With Support from Former Tampa Bay Buccaneer Mike Liedtke

Personalized and targeted medicine for rare lung and other cancers

Protheragen announced its disease model development platform to develop 2D cell culture, 3D organoid, and animal models for researchers.

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

DOVER, Del. — The Delaware Rare Disease Advisory Council held its inaugural meeting on Tuesday, taking its first steps to address innovation and research, patient care, and affordable and accessible treatment therapies.     Delaware is one of 27 states with a Rare Disease Advisory Council. Established by Senate Bill 55, sponsored by Sen. Nicole Poore […]

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.

Union Minister of State for Health and Family Welfare, Anupriya Singh Patel, announced that up to Rs. 50 lakhs per patient has been allocated for the

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)

A five-year-old boy in New York state has died from a rare inflammatory disease believed to be caused by the new coronavirus, Governor Andrew Cuomo said Friday.

NEW YORK: A five-year-old boy in New York state has died from a rare inflammatory disease believed to be caused by the new coronavirus, Governor Andrew Cuomo said on Friday.“There have been 73 reported cases in NY of children getting severely ill with symptoms similar to Kawasaki disease...

PTC Therapeutics has reached an agreement to acquire Censa Pharmaceuticals and its lead asset, an experimental metabolic disorder therapy that is now being prepared for a pivotal study. The deal, announced after the market close Wednesday, will bring South Plainfield, NJ-based PTC (NASDAQ: PTCT) another compound for its pipeline of rare disease drugs. Wellesley, MA-based […]

Five-year-old Ollie Williams went down suddenly by a rare disease called Stevens-Johnson syndrome this May. This disease caused a sudden outburst of blisters

New gene that causes melorheostosis, a rare group of conditions involving painful and disfiguring overgrowth of bone tissue, is identified by the team

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.