Interest in lysosomal storage diseases (LSDs) has increased significantly, because several new therapeutics received FDA approval in 2003. Though long overlooked by most major pharmaceutical ...

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...

Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...

The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

Julien Grogg, PhD, MBA - CEO of Release Therapeutics Release Tx logo Dr. Julien Grogg will Lead the Company's Transition to Clinical ...