by Peter Ciszewski | Nov 24, 2018
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Krabbe disease (also called globoid cell leukodystrophy), a degenerative disorder that affects the nervous system. It is caused by...
by Peter Ciszewski | Nov 23, 2018
The FDA granted accelerated approval to Venetoclax (Venclexta) in combination with azacitidine, decitabine or low-dose cytarabine to treat newly diagnosed patients with acute myeloid leukemia (AML) aged 75 years or older. The approval also applies to adults with AML...
by Peter Ciszewski | Nov 21, 2018
Daniel de Boer, Founder and CEO of ProQR, discusses Leber’s congenital amaurosis (LCA), the most common genetic cause of childhood blindness that affects about 15,000 patients in the Western world. LCA leads to poor vision and blindness for which there is...
by Peter Ciszewski | Nov 20, 2018
The U.S. Food and Drug Administration (FDA) approved Gamifant (emapalumab-lzsg), an interferon gamma (IFNγ) blocking antibody for the treatment of patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or...
by Peter Ciszewski | Nov 20, 2018
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the...
