TEL AVIV, Israel, Sept. 26, 2016 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, today announced that Zohar Argov, MD, Special Medical Advisor to the Executive Chairman of Bioblast Pharma, will present an invited lecture on “Trehalose in Oculopharyngeal Muscular Dystrophy (OPMD)” at the annual scientific meeting of the Muscle Study Group Society (MSGS) to be held today in Snowbird, Utah. The MSGS is an interdisciplinary US-based group devoted to the development of treatments for neuromuscular disorders.
The lecture will summarize the general approach that Bioblast is taking in the use of trehalose as a potential therapeutic agent for OPMD. The topics to be discussed include:
About Professor Argov
Professor Zohar Argov, MD, is world renowned for his studies and discoveries in myology and neurological diseases. He is Professor (Emeritus) of Neurology & Josephine Frank Kanrich Chair of Neuromuscular Diseases at Hebrew University Hadassah Medical School, Jerusalem, Israel. Professor Argov is also an Adjunct Professor at the Department of Neurology/Neurosurgery, Montreal Neurological Institute, McGill University, Montreal, Canada. He was President of the European Neurological Society and Chairman of the European Neurological Society Muscle & Neuromuscular Disorders Subcommittee. Prof. Argov currently serves on the executive committee of the World Muscle Society.
Professor Argov, a board-certified neurologist, earned his medical degree from Hebrew University Hadassah Medical School in Jerusalem, Israel.
About Trehalose 90mg/mL IV Solution
Trehalose 90mg/mL IV solution is a chemical chaperone that protects against pathological processes in cells. It has been shown to reduce pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation as well as acting as an autophagy enhancer. Trehalose 90mg/mL IV solution has been documented as demonstrating significant promise in preclinical animal models of OPMD and other PolyA/PolyQ diseases.
In OPMD, trehalose 90mg/mL IV solution is being developed to prevent the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death.
About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing), eyelid drooping (ptosis), the loss of muscle strength, and weakness in multiple muscles of the limbs. Symptoms generally appear in mid-life and get worse over time. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, and may suffer from repeated incidents of aspiration pneumonia. Aspiration pneumonia and severe emaciation may result in death. The disease is caused by a genetic mutation responsible for the creation of a mutant protein (PABPN1) with an expanded polyalanine domain that aggregates within patient muscle cells. There is currently no approved pharmacologic treatment for OPMD.
About the Muscle Study Group Society
The Muscle Study Group Society (MSGS), is a consortium of scientific investigators from academic and research centers who are committed to the cooperative planning, implementation, analysis and reporting of controlled clinical trials and of other research for muscle and other neuromuscular diseases. The MSGS aims to advance knowledge about the cause(s), pathogenesis, epidemiology, and clinical manifestations of muscle disease and related neuromuscular disorders and to develop and implement strategies to examine promising therapeutic interventions.
About Bioblast Pharma Ltd.
Bioblast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, with a lead drug candidate -- trehalose 90mg/mL solution -- in Phase 2 development. Bioblast was founded in 2012 and is traded on the NASDAQ under the symbol “ORPN”. For more information, please visit our website, www.bioblastpharma.com, the content of which is not incorporated herein by reference.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. Historic results of scientific research and clinical and preclinical trials do not guarantee that the conclusions of future research or trials would not suggest different conclusions or those historic results referred to in this press release would not be interpreted differently in light of additional research and clinical and preclinical trial results. Because such statements deal with future events and are based on Bioblast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Bioblast Pharma could differ materially from those described in or implied by the statements in this press release, including those discussed under the heading "Risk Factors" in Bioblast Pharma's Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC") on March 29, 2016, and in any subsequent filings with the SEC. Except as otherwise required by law, Bioblast Pharma disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.GlobeNewswire
Last updated on: 27/09/2016
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