Latest Glycogen storage disease type II Stories

DOYLESTOWN, Pa., May 14, 2013 /PRNewswire/ -- Callidus Biopharma, Inc., a development-stage biotechnology company focused on creating breakthrough biologic drugs for a range of orphan diseases, announced today that it has closed on $4.6 million in Series A financing led by two strategic investors. The Company intends to use the proceeds to accelerate pre-clinical development of its drug discovery pipeline of therapies for lysosomal storage diseases (LSDs), including lead candidates...

LONDON, April 27, 2012 /PRNewswire/ -- Based on its recent analysis of the piston and plunger pumps market, Frost & Sullivan recognises Peroni Pompe S.p.A. with the 2012 European Frost & Sullivan Customer Service Leadership Award. "The company's strong brand name recognition in the market can be attributed to the superior customer services it provides to its clients," notes Frost & Sullivan Research Analyst Niranjan Paul. "Through the effective leverage of its customer...

Researchers will use computer modeling and patient surveys to study health and economic outcomes for screening of Krabbe disease, Pompe disease, and phenylketonuria ANN ARBOR, Mich., March 1, 2012 /PRNewswire-USNewswire/ - University of Michigan researchers will use a $2.5 million grant from the Agency for Healthcare Research and Quality to study long-term health outcomes and cost-effectiveness of newborn screening. This study, led by Dr. Lisa A. Prosser, Ph.D., M.S., could...

Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, but some children with Pompe disease produce an immune reaction that blocks the benefits of the life-saving enzyme treatment. To date there has been no success in eliminating or suppressing this immune response. Now research led by Duke University Medical Center, with...

CHICAGO, Dec. 19, 2011 /PRNewswire/ -- On this week's episode of "The Ben Heck Show," exclusively sponsored by element14, Ben receives a request from a teacher in New Jersey to create an accessible controller for a student living with Pompe disease, a neuromuscular disorder causing muscle weakness. With Ben's mod, 13-year-old Patrick Crowley will be able to play video games requiring complex button sequences, such as X-Men Origins: Wolverine(TM). (Photo:...

(Ivanhoe Newswire) -- Researchers set their eyes on a new treatment! Patients that have been affected by melanoma of the eye, ocular or uveal melanoma, die within an average of two to four months once the disease has spread to the liver due to ineffective treatment.  Only about one in ten patients live for a year.  A recent study has found that with a new treatment patients may be able to reduce the growth of the disease. James Pingpank, associate professor of surgery at the University...

NOVATO, Calif., July 28, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis. Patients originally enrolled in the initial Phase 1/2 study of GALNS (MOR-002) were continued on therapy in a new extension study (MOR-100). In the MOR-100 study, patients were treated at a 2.0 mg/kg/week dose and have been followed for an additional 24 weeks....

CRANBURY, N.J., May 4, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company at the forefront of developing therapies for rare diseases, today announced financial results for the first quarter ended March 31, 2011. The Company also highlighted recent and upcoming milestones, including an update on patient enrollment in the ongoing Phase 3 study of its lead program Amigal(TM) (migalastat HCl) for Fabry disease. Development Pipeline Highlights Enrollment in...

SAN DIEGO, April 7, 2011 /PRNewswire/ -- Zacharon Pharmaceuticals, Inc. today announced that the company has entered into a strategic research collaboration with Pfizer Inc. to develop drugs for orphan diseases, including lysosomal storage disorders. The potential value of the collaboration to Zacharon is approximately USD $210 million. The collaboration includes the potential development of compounds that may be discovered using Zacharon's innovative platform for developing small molecule...

CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease. Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase 2...