Qure's AMT-130 shows 80% Huntington's Disease progression slowing. Click to read more on QURE's financially strong, advancing ...

Another reason for choosing a particular modality is that it is a team’s specialty. Strategic choices can be less about ...

Treatments for COPD, psoriasis, and neuroendocrine tumors, as well as a next-generation COVID-19 vaccine are under review.

Insurer EmblemHealth is leading a class-action lawsuit against a subsidiary of AstraZeneca, claiming that the company blocked ...

I care deeply because this chronic disease is mine. It lives inside me. It’s radically changed my life and the life of my ...

SynaptixBio was named the UK’s Most Innovative Rare Disease Biotech Company 2025 at the Global Health and Pharma ...

“Our CB-SCs can target different compartments of the immune system, educating and correcting their dysfunctions at a fundamental level,” says Dr. Zhao. “This therapy has the potential to revolutionize ...

Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~~ ...

Glycomine raises $115M Series C to advance GLM101, a mannose-1-phosphate therapy for PMM2-CDG rare disease, toward Phase 2b ...

Glycomine’s Series C raise highlights investor confidence in the company amid pressure on the paediatric rare disease sector.

ATSN-201 was given regenerative medicine advanced therapy designation to treat X-linked retinoschisis, for which there ...