Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters
Giroctocogene fitelparvovec study meets primary and key secondary objectives of superiority compared to prophylaxis
A one-time dose of DURVEQTIX has reduced bleeds post-treatment compared to standard of care with a median annualized bleed rate (ABR) of zero bleeds (range 0 to 9.9)
The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment
Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant
The survey data presented in “The State of the Biopharmaceutical Industry 2024” report reveals that N=21 (18%) of industry professionals were convinced that CGT will dominate as the most important trend in pharma next year
Laurus will also provide funding for the clinical trials and will launch these products in India and emerging markets
IIT Kanpur pioneers ground-breaking gene therapy technology for Hereditary Eye Diseases
The new center will focus on supporting the clinical trials clients who are developing cell and gene therapies
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