Today, the U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very ...

Title: Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program Presentation Number: 10 – Poster Session Date: Saturday, ...

Vitamin B12 is essential for brain health, which affects everything from memory to mood. Let's learn why B12 is important for the brain and how to make sure you're getting enough of it.

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...

The enzyme replacement therapy (ERT) market is projected to grow significantly, rising from US$ 10,707.6 million in ...

In India, 32 patients, diagnosed with this condition by the Centres of Excellence (CoEs), have registered themselves with the ...

Researchers identified genetic variants affecting sucrase, a key digestive enzyme, linked to IBS risk and symptom severity.

A strong immune system is your body's natural defence against infections and diseases. One of the best ways to keep it ...

Fabry Disease Clinical Trial Pipeline Major pharmaceutical companies are working to advance the pipeline space and unlock the future growth ...

Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...