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February 24, 2025
Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.
February 21, 2025
Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.
February 18, 2025
Tenosynovial giant cell tumor (TGCT) is rare and non-malignant, forming in or near joints, but which if left untreated can cause damage to joints and surrounding tissue, potentially leading to significant disability.
February 17, 2025
The agency has received certification with the Eco-Management and Audit Scheme for its commitment to environmental sustainability.
The approval makes Evrysdi the first and only tablet for treating spinal muscular atrophy.
The pledge signifies ArisGlobal’s commitment to upholding high standards and best practices for the responsible use of artificial intelligence.
Agnes Cwienczek, director of Product Management at ArisGlobal, talks with Pharmaceutical Technology® about how careers for women in STEM fields can be nurtured.
February 14, 2025
In this exclusive Drug Digest video interview Anil Kane from Thermo Fisher Scientific will be tackling the topic of advances in small-molecule manufacturing and several other experts will provide brief commentaries on associated topics.
February 13, 2025
With the approval, FDA granted SpringWorks a rare pediatric disease priority review voucher.
February 12, 2025
Acoramidis was approved by FDA in November 2024 and is prescribed in the US under the brand name Attruby.