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News Archive
The U.S. Food and Drug Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
"Many people are now saying: 'Is it really that easy? Then why didn't anyone think of it a long time ago?' But you have to have the right idea at the right time," says Annette Röttger, physicist at the Physikalisch-Technische Bundesanstalt, in a pleased way. Annette Röttger and her scientific team managed to do something that was previously thought to be impossible: they developed a primary standard for the measurement of short-lived radioactive thoron.
Defects in the gene that encodes the XIAP protein result in a serious immune malfunction. Scientists used biochemical analyses to map the protein's ability to activate vital components of the immune system. Their results have recently been published in Molecular Cell, a journal of international scientific repute.
Many patients undergoing lumpectomy surgery at NYU Langone Medical Center for the removal of an early detected breast tumor - the surgical option of choice for this diagnosis - are benefitting from new intra-operative technology that detects microscopic amounts of cancer cells on removed tumor tissue not visible during or following surgical intervention.
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