07:00 , May 21, 2015 |  BC Innovations  |  Targets & Mechanisms

Intersection in marrow

An unlikely connection between Gaucher's disease and multiple myeloma has been uncovered by a U.K. group that found a common node in the pathways responsible for the bone disease symptoms that accompany both diseases. Suppression...
07:00 , May 4, 2015 |  BioCentury  |  Emerging Company Profile

Nixing Niemann-Pick

Vtesse Inc. is hoping to change the course of disease in Niemann-Pick type C, a fatal ultra-Orphan lysosomal storage disorder, using a compound plucked from NIH's Therapeutics for Rare and Neglected Diseases repurposing project. Niemann-Pick...
07:00 , Oct 2, 2014 |  BC Innovations  |  Strategy

Progressive thinking in MS

Despite the slew of treatments that have entered the market in the last few years for relapsing-remitting multiple sclerosis, there have been few advances in the progressive form of the disease, largely because there is...
08:00 , Feb 20, 2014 |  BC Innovations  |  Distillery Techniques

Technology: Assays & screens

Approach Summary Licensing status Publication and contact information Assays & screens Diagnosing and monitoring treatment responses in patients with Niemann-Pick disease type C1 (NPC1) using fluorescence-based volumetric measurement of lysosomes A fluorescence-based assay in B...
08:00 , Feb 13, 2014 |  BC Innovations  |  Targets & Mechanisms

A RIPping target in Gaucher's disease

Enzyme-replacement therapies are highly effective for most patients with Gaucher's disease but do little for those with the neurological childhood forms of the disease. Now, an Israeli and U.K. team has unlocked the mechanism of...
07:00 , Apr 9, 2012 |  BC Week In Review  |  Clinical News

Brazaves miglustat regulatory update

Japan's Ministry of Health, Labor and Welfare (MHLW) approved Actelion's Brazaves miglustat to treat Niemann-Pick type C disease. Miglustat is marketed as Zavesca for the indication in 42 countries including Australia, Brazil, Canada, Russia, South...
07:00 , Apr 9, 2012 |  BioCentury  |  Finance

Highlights of weekly biotech stock moves

Actelion Ltd. (SIX:ATLN) was up CHF0.36 to CHF33.36 last week after Japan's Ministry of Health, Labor and Welfare (MHLW) approved Brazaves miglustat to treat Niemann-Pick type C disease. Separately, Actelion discontinued development of setipiprant. The...
08:00 , Nov 7, 2011 |  BioCentury  |  Emerging Company Profile

Orphazyme: One for all

Drug development for lysosomal storage disorders has largely centered on enzyme replacement therapies, which yield one drug per disease. Orphazyme ApS believes its recombinant heat shock protein 70 (Hsp70) and small molecules that induce heat...
07:00 , Oct 11, 2010 |  BC Week In Review  |  Clinical News

Tracleer bosentan regulatory update

FDA issued a warning letter to Actelion after an inspection revealed that the company failed to comply with postmarketing safety reporting requirements for 3 drugs. FDA said the company failed to report serious and unexpected...
07:00 , Sep 6, 2010 |  BioCentury  |  Strategy

Laying siege

sanofi-aventis Group made clear in its conference call last week that it's in no hurry to complete its proposed acquisition of Genzyme Corp. - with the obvious implication that time is on its side. If...