Neuroendocrine tumors (NETs): The complex group...

Alnylam’s Onpattro Receives First-Ever US FDA Approval for Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults US FDA approved Alnylam’s Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneurop...

Astellas’ Ophthalmology Innovation to Strengthen Further With Quethera Acquisition Astellas has acquired Quethera, a gene therapy company headquartered in the UK, which is focused on developing novel treatments for ocular disorders, such as glaucoma. Through this transaction, Astellas has acquired Quethera’s ...

Sarepta builds in gene therapy with $30M Lacerta deal Sarepta has dampen its enthusiasm for an ongoing gene therapy candidate. Sarepta has made a tie-up with lacerta that will add 3 more programs to 8 already existing and are ready to expand its focus beyond muscular dystrophy. Sarepta has committed to pay Lacerta ...

Progenics got FDA Approval on Iobenguane I 131 for Rare Adrenal Gland Tumors  A good news for unresectable rare adrenal tumor  patients as oncologists now have a new treatment to offer . On July 30, 2018, the Food and Drug Administration approved Progenics Pharmaceuticals iobenguane I for adult and pediatric patient...

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) – a lesser developed window Mucopolysaccharidosis (MPS) is a group of rare, hereditary and incurable “storage diseases”, which is named after mucopolysaccharides (sugars bound to proteins). The stored mucopolysaccharides are called as glycosaminoglycans (GAGs). ...

Niemann-Pick disease type C (NPC) is a lipid storage disease that can present in infants, children, or adults. Neonates can present with ascites and severe liver disease from infiltration of the liver and/or respiratory failure from infiltration of the lungs. The classic presentation occurs in mid-to-late childhood ...