The reduction of heparan sulfate, the key biomarker of disease pathology in MPS IIIB, at 30 days post gene transfer indicates early and robust systemic delivery of ABO-101, and further supports our planned approach using intravenous delivery of ABO-101 in the treatment of Sanfilippo
syndromes," stated Timothy J.
BioMarin Pharmaceutical announced that it presented interim data from a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase with a peptide derived from insulin-like growth factor 2, for the treatment of Sanfilippo
B syndrome or mucopolysaccharidosis IIIB at WORLDSymposium 2018.
& Son, in partnership with Damen Jackson, has received a "Best Use of Packaging" Silver Award in the 2017 Transform Awards for its Fisher Chefs Naturals packaging redesign.
said the acquisition of the Squirrel Brand business provides an established customer base and branded product line that will help facilitate the execution of the company's expanding consumer reach growth strategy, especially in alternative distribution channels.
Small Molecule for Sanfilippo
Syndrome - Drug Profile 53
M2 PHARMA-September 7, 2017-BioMarin Reports on Study of BMN 250 for Treatment of Sanfilippo
B Syndrome (MPS IIIB) at ICIEM 2017
of Affordable Dumpsters denied allegations his company was circumventing local waste disposal regulations.
University of Manchester and Central Manchester University Hospital NHS Foundation Trust (CMFT) researchers have developed the pioneering approach for Sanfilippo
disease (also known as mucopolysaccharidosis type III or MPS III).
The four-year-old has Sanfilippo
disease, an incurable illness which will destroy his brain functions and physical ability.
, professor of obstetrics, gynecology, and reproductive sciences at the University of Pittsburgh and director of the division of reproductive endocrinology and infertility at MageeWomens Hospital in Pittsburgh.
In his new role, Sanfilippo
heads the company's operations, Technology and Enterprise Risk Management functions.
He participated in a 2 year medical research study at the University of Minnesota to get medicine approved for his rare illness Sanfilippo
Syndrome (MPS IIIa).