According to an estimate by the Centers for Disease Control and Prevention (CDC), SCD affects over 100,000 Americans and is a major public health concern as it resulted in 75,000 hospitalizations between 1989 through 1993, and cost over $475 million. The Medical expenditure of children with...
AzurRx BioPharma (NASDAQ: AZRX), a development stage company that is developing oral, nonsystemic, recombinant proteins for the treatment of gastrointestinal diseases, announced the results from a Phase II Clinical Trial of MS1819. The phase II trial named OPTION was an open-label, crossover trial being carried...
CRISPR, which is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary genome editing approach that helps change an individual’s DNA, by altering it at a specific location. The Crisper-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,...
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...
Cystic Fibrosis is a chronic genetic disorder of the mucus glands that affects 2,500 newborns each year in the U.S. 30,000 people are currently affected by this disease and about 1 in 20 or roughly 12 million Americans are carriers of the “CF” gene and are unaware of...
