Earlier this week, Epygenix Therapeutics shared that the first Canadian participant was dosed in their latest clinical trial

Patient registries are an increasingly important medium in the rare disease research and development environment.

Chimeric antigen receptor T-cell (CAR-T) therapies have demonstrated significant efficacy against certain blood cancers such as acute...

Just last month, the European Medicines Agency (EMA) granted an orphan drug designation for a treatment against pyridoxal...

Kalydeco, developed by Vertex Pharmaceuticals, was first approved by Health Canada in 2012 as the first treatment available.

A review process of the European Orphan Medicines Regulation is underway. This regulation is specific medicines for rare diseases.

New pacts for health emerge in the public discourse in the post-pandemic period, but they still need to become reality.

On March 10th and 11th of 2022 a group of interdisciplinary social science researchers met face-to-face and on-line to share their...

It has become more familiar in the last two years to hear about access to medicines - namely access to vaccines or expected drugs against...

In the Brazilian scenario, patients with rare diseases must continue to litigate in court for the equitable distribution of health.

About one month ago, Avrobio announced that it was deprioritising the Fabry gene therapy program as a result of various considerations.

The Canadian Federal Health Minister, Jean-Yves Duclos announced that the regulatory amendments from the Patented Medicine Prices Review...

It is widely understood by clinical care communities and health authorities that we need to identify better ways of using scientific data.

On November 12th, 2021 Health Canada announced that it would grant market approval for Sanofi’s new treatment for Pompe disease.

We recently heard about a captivating documentary, based on the lived experience of a patient, named Mitch, with a rare disease.

Fast’ is not usually an adjective readily associated with developing drugs. This may soon change - at least in the Netherlands.

On April 19th, 2021, the Liberal government tabled the federal budget. This was the first budget proposed in two years as a result of the...

Patient registries present an exceptional opportunity to address the needs of rare diseases through data collection and research.

The Canadian research team's response to the call for Online Engagement on a National Strategy for Drugs for Rare Diseases

The recognition of the right to health in Brazil, given by the Federal Constitution of 1988 (CF 88), is unequivocal and outlined with...