Fabry disease is a lysosomal storage disease arising from a deficiency of the enzyme α-galactosidase A (GLA). The enzyme deficiency results in an accumulation of glycolipids, which over time, leads to cardiovascular, cerebrovascular, and renal disease, ultimately leading to death in the fourth or fifth...
Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to...
A great chef could create an outstanding dish that revolutionizes cuisine – every time they prepare the recipe for two, it may come out perfect and impress everyone who tries it. However, they would probably struggle to reproduce the same show-stopper dish with the same...
There are already more than 50 nanomedicine formulations in the market and over 400 currently in clinical trials, thanks to the unique versatility of these therapeutics compared to conventional drugs. Several European projects are developing nanomedicines in areas with unmet medical needs, such as Cupido...
It is our pleasure to inform you that (virtual) oral communication under the tittle “Scale-up and Quality by Design application to the robust production of an enzyme-loaded liposomal formulation by DELOS-susp method” has been accepted for the 14th Mediterranean Congress of Chemical Engineering that will...
ETPN2020 is the 15th annual event of the ETPN – European Technology Platform on Nanomedicine, to be held on Oct. 14-15, 2020, as a virtual event this year in the context of the COVID-19 pandemic. “Translational issues in nanomedicine” Workshop will be held in the context...
In this occasion the "Marató de TV3: Minories que fan una Majoria", is devoted to rare diseases. La Marató de TV3 is an annual telethon broadcast by Televisió de Catalunya and the Fundació La Marató de TV3 to raise funds for scientific research into diseases...
On the 27th October, our colleague Judit Tomsen form ICMAB-CSIC-CIBER presented the advances for Fabry disease treatment obtained in the context of Smart4Fabry project at the XIII Science Festival in Barcelona. This event inclues more than 200 activities, and is intended to get Science and...
Dolores Bueno from ICMAB-CSIC-CIBER will give the talk Show your rare!, which will be focused on rare disease, with a view of Fabry and Smart4Fabry approach and the proposed solutions. The event can be followed in real time through the event web page https://naukas.com/2019/09/19/en-directo-naukas-bilbao-2019/...