Enzyme Replacement Therapy Market Size, Growth & Forecast By 2033
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 ...
precisionmedicineonline2d
UniQure Testing Higher Gene Therapy Dose in Phase I/II Fabry Disease Trial
The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.
Zevra Therapeutics to Present at the 21st Annual WORLDSymposium™
Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposiumEight abstracts discussing data and clinical ...
Release Therapeutics Announces New CEO To Advance Groundbreaking Treatment For Brain Disorders
Julien Grogg, PhD, MBA - CEO of Release Therapeutics Release Tx logo Dr. Julien Grogg will Lead the Company's Transition to Clinical ...
The Financial Express6d
Rare disease patients urge PM Modi to address delay in Rs 974 crore national programme roll-out
According to the open letter, the delay in rolling out the proposed ₹ 974 crore national programme on rare diseases has exacerbated these challenges, leaving patients, most of whom are children, in ...