Fierce Pharma

Travere maps course for Filspari's $3B US opportunity after landmark rare disease nod

With the path to blockbusterland now in sight, Travere Therapeutics is ready to hit the ground running after Monday’s ...

Veld Pharmaceuticals GmbH Announces Collaboration with Chiesi Farmaceutici S.p.A to Expand Access to Rare Disease Therapies Across Africa

Veld Pharmaceuticals GmbH (“Veld”), a Switzerland-based pharmaceutical access and services company, today announced a collaboration with Chiesi Farmaceutici S.p.A (“Chiesi”) to expand patient access ...

UKHSA warning after 1,500 cases of rare disease confirmed

There are around 1,500 laboratory-confirmed cases of Lyme disease in England and Wales each year, with a further 1,000 to ...

Rare disease advocates push for local insurance reforms, improved access to care

Odds are, most people have probably met someone with a rare disease — or even has one themself. While each condition on its own might be uncommon, altogether there are about 25-30 million people in ...
Regulatory Affairs Professionals Society

Høeg touts FDA efforts to speed rare disease drugs to market

The top drug regulator at the US Food and Drug Administration (FDA) said the agency is working to develop new pathways for ...
Business Insider

Palvella Therapeutics Appoints John D. Doux, M.D., M.B.A., Dermatologist and Recognized Leader in Rare Skin Diseases, to Board of Directors

Physician and seasoned life sciences investor with more than two decades of experience across clinical practice, ...

This rare disease destroys a child's ability to walk and speak. Could a new drug help?

After a string of denials for rare disease treatments, the FDA greenlit a drug for Hunter syndrome, a progressive illness ...

A mom and tech entrepreneur building AI advocate for rare-disease families like hers

AI-powered healthcare startup Citizen Health is using the technology to focus on rare disease, helping manage patient lives ...

Artificial Intelligence (AI) Platforms Could Enhance Jaguar Health’s Development and Anticipated Commercialization of Crofelemer for Rare Disease Intestinal Failure (IF) Programs

Global Market for Short Bowel Syndrome, Including Intestinal Failure (IF), Estimated to Reach $8.0 Billion by 20331AI ...