CAMBRIDGE, Mass.–(BUSINESS WIRE)–Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today four-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the … Continue reading
Over the years many cell and gene therapy approaches have been tested in animal models of lysosomal storage diseases, and two main approaches have emerged as the most promising for translation into human clinical trials: In vivo gene transfer by direct infusion of viral vectors (AAV and lentivirus vectors) encoding normal enzymes; or modification of bone marrow stem cells in culture with viral vectors encoding normal enzymes (retrovirus and lentivirus vectors) followed by transplantation (this latter approach is often referred to as ex vivo gene therapy). Continue reading
A drug called GM1, being tested to treat Parkinson’s disease and related neurodegenerative maladies, has successfully restored motor skills in an animal model of Huntington disease during laboratory preclinical testing, at least for a few weeks after the dosing stopped. GM1, a molecule produced in the body, is a type of lipid and is part … Continue reading
What is MPS VI? MPS VI (mucopolysaccharidosis VI), also known as Maroteaux-Lamy Syndrome, is an inherited lysosomal storage disorder caused by the deficiency of arylsulfatase B, an enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). If the enzyme is not present in large enough amounts, the normal breakdown of … Continue reading
If you have a spare $4000 laying around, you can get the full copy of this business intelligence report. If not, we have to be content with this press release. I’m assuming that a variety of pharma companies are buying it. I also don’t think some of the statements (in this summary, at least) take … Continue reading
To see the full press release: Charities unite worldwide to fund Sanfilippo research Led by the Team Sanfilippo Foundation, a group of worldwide charities has announced a $145,000 (AUD) grant to Dr Kim Hemsley and Professor John Hopwood in Adelaide, Australia to study the fundamental disease processes involved in the pathology of MPS III, also … Continue reading
The Korean pharmaceutical company Green Cross has developed and received approval from the Korean FDA for an alternative, licensed therapeutic enzyme to treat Hunter Syndrome, a rare disease that until now had only one treatment option available.
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Genistein in Sanfilippo disease: A randomized controlled crossover trial. Authors de Ruijter J, Valstar MJ, Narajczyk M, Wegrzyn G, Kulik W, Ijlst L, Wagemans T, van der Wal WM, Wijburg FA. Journal Ann Neurol. 2012 Jan;71(1):110-20. doi: 10.1002/ana.22643. Affiliation Department of Pediatrics and Amsterdam Lysosome Centre “Sphinx”, University of Amsterdam, Amsterdam, The Netherlands. Abstract OBJECTIVE … Continue reading
Here’s the latest: Researchers at Queen Mary, University of London believe that omega-3 fatty acids can protect nerves from being injured and help regenerate the ones that are harmed, based on preclinical research with mice. Omega-3s, of course, are found in fish oil. And beyond the latest finding, scientists are discovering that fish oil has the potential to solve plenty of health problems. Continue reading
The clinical trial plan for the U.S. natural history study for Sanfilippo Syndrome Type B has just been published in clinicaltrials.gov. It is essentially the same plan as the current Natural History study for Type A. We have reproduced it here and linked to the official clinical trials record. This is further evidence that Shire is proceeding rapidly with a plan to carry out clinical trials of enzyme replacement for MPS 3B.
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Press release from Marketwatch, January 5, 2012. MPS 7 Patients May at Last have an Opportunity for Enzyme Replacement Therapy NOVATO, Calif., Jan. 5, 2012 /PRNewswire via COMTEX/ — Ultragenyx Pharmaceutical, Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has in-licensed an enzyme replacement therapy program … Continue reading
Dublin-based Shire’s ($SHPGY) Human Genetic Therapies unit and venture capitalists from Atlas Venture have formed a multiyear alliance to hunt for new investments in the ripe field of treating rare diseases, Cambridge, MA-based Atlas revealed. For their part in the collaboration, Atlas’ partners plan to use their years of experience in starting new biotech companies. … Continue reading