• Latest Posts
Interview 3 Mar 2023

Interview: how has the first gene therapy for hemophilia B transformed research?

European Commission approves first gene therapy for hemophilia B

GC Biopharma acquiring hemophilia rare disease pipeline

Opinion 28 Feb 2023

Life changing treatments for rare disease – a beacon of hope

5 advancements in rare disease research over the past year

ADVERTISEMENT

10 biotech companies making a difference in rare diseases

Beyond Biotech podcast 35: Rare Disease Day

Moderna and Life Edit Therapeutics look to speed up gene editing therapies

First patient enrolled in Pharming APSD trial  

FDA approves alpha-mannosidosis drug

Positive results for INOVIO recurrent respiratory papillomatosis trial

ADVERTISEMENT