The therapy is a critical treatment option for various life-threatening disease. Earlier, GST on gene therapy was set at 12 ...

The landscape of rare diseases is constantly changing as new ailments and conditions are identified and reported regularly.

The government may slash duties on imported rare disease and cancer drugs in the Union budget aiming to make lifesaving treatments affordable and ease financial burdens on patients ...

On a recent visit to Washington DC, Ho sat down with GEN Editorial Director Kevin Davies, PhD, to discuss Denali’s progress ...

We might be remembered in a warm paragraph in his memoirs. There was a second part to the worry; Werner was born with Gaucher ...

Genzyme anticipates supply constraints of Cerezyme (imiglucerase), a treatment for Gaucher disease, and Fabrazyme (agalsidase beta), used to treat Fabry disease, while the facility shuts down for ...

The trailblazers – Genzyme’s Henri Termeer chief among them with development of the first therapy to treat Gaucher disease – quickly recognised the vital role of external stakeholders to get ...

9, 1964, in Hot Springs, the oldest child of Ann and Dr. John Trieschmann. He was born with Gaucher's disease, an enzyme disorder that causes skeletal abnormalities. He graduated from Lakeside ...

In addition to general challenges, there can be specific aspects of the disease and its management that can impact treatment adherence. In some rare conditions, for example Gaucher’s disease, ...

Limitation of down gaze is a feature of Gaucher disease, which is most obvious when walking down stairs manifesting as unsteadiness and also compensatory head-thrusting. In the late infantile/juvenile ...

β-Glucocerebrosidase, an enzyme for replacement therapy in Morbus Gaucher treatment, is obtained from carrot ... 2017), along with the moss-GAA (acid alpha-1,4-glucosidase) against Pompe disease ...