Health Canada has approved a new drug treatment for adults living with Fabry disease, a rare genetic disorder that can affect multiple organs and impact the quality of life.

Elfabrio® (pegunigalsidase alfa for injection) is an enzyme replacement therapy indicated for the longterm treatment of adult ...

Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...

India’s rare disease care relies on crowdfunding as costly therapies outstrip public support. Government aid is capped and ...

A parliamentary forum has urged urgent action on funding shortfalls and treatment delays for patients with Lysosomal Storage ...

The Indian Medical Parliamentarians’ Forum (IMPF), a cross-party collective of 45 medical professionals turned ...

The Indian Medical Parliamentarians Forum (IMPF) — ?a cross-party group of 45 MPs who are also medical professionals — ?has ...

The Indian Medical Parliamentarians’ Forum (IMPF), a group of 45 members of parliament who are also medical professionals, ...

The FDA approves the first gene therapy for a rare immune disorder, a landmark decision for genetic medicine, treatment ...