Category Archives: Drugs

Big Business Potential in Myotonic Dystrophy Drugs

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My most recent blog post on the “Race for the Cure” For Myotonic Dystrophy drugs and a potential cure had stated that the market size that is reachable for this disease is between 2-5 Billion dollars. Today a recent Wall St. Journal Article “The Big Business of Orphan Drugs” confirms that the market for These type of Orphan Drugs is extremely lucrative for companies and patients alike. Read about Small is Lucrative for Drug Companies. The Wall St. Journal article of course is not available without a Subscription.

The Wall street journal reported that incentives from the FDA to develop so called orphan drugs can mean quicker and more certain approval, tax benefits for the developer, and seven years of protection from competition after approval. Orphan drugs are defined as experimental treatments for disease with fewer than 200,000 patients at one time.

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Researchers Race for Myotonic Dystrophy Cure

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Race for the Cure for Myotonic Dystrophy

Race for the Cure for Myotonic Dystrophy

Myotonic Dystrophy may be the first of the muscular Dystrophy to have a definitive cure established. In the last few years researchers have isolated the issue they believe causes myotonic dystrophy. In the last couple of years therapies that have targeted this problem have reversed the disease in cell and animal models. This has set up a race now for researchers to refine the therapeutic compounds and begin with human trials to see if the animal to human therapeutic transfer works as it is believed to. Thus, this would lead to the first potential cure for a type of muscular dystrophy.

The Myotonic Dystrophy Foundation (MDF) in Menlo Park, CA  has taken a lead in assisting researchers with moving forward with this disease elimination process with funding and assistance in establishing a patient registry. The MDF was the early funder of a new Research Collaborative effort lead by Charles Thorton out of the University of Rochester. The foundation has also  been instrumental in educating patients and physicians in this complex and slowly progressing disease that results in premature death. A patient conference is planned for early November of 2013 in Houston, TX

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Prosensa presenting at JP Morgan Healthcare Conference

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This is a press release if there is any additional information we will share this with you.

Prosensa to Present at J.P. Morgan Healthcare Conference

LEIDEN, The Netherlands, Jan 03, 2013 (BUSINESS WIRE) — Prosensa, the private Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that Hans Schikan, Chief Executive Officer, will be presenting at the 31st Annual J.P. Morgan Healthcare Conference in San Francisco.

Prosensa’s presentation is scheduled at the Westin St. Francis on Union Square at 4.30pm PST on Monday, January 7, 2013 and will provide a corporate update to the investment community. Prosensa’s lead compound (drisapersen), being developed by GlaxoSmithKline, is currently in late-stage phase III clinical trials for Duchenne muscular dystrophy.

–ENDS–

Notes to editors:

About Prosensa

Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy and Huntington’s disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound (drisapersen), being developed by GSK, is currently in late-stage phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit www.prosensa.com .

Prosensa won the 2012 Emerging Star Award at the European Mediscience Awards and was listed as a Fierce 15 Biotech Company.

SOURCE: Prosensa

        
        Prosensa enquiries: 
        Luc Dochez +31 71 332 2085 
        or 
        Hans Schikan +31 71 332 2100 
        Media enquiries: 
        College Hill Life Sciences 
        Melanie Toyne-Sewell / Anastasios Koutsos / Henry Stanley 
        +44 20 7457 2020 
        or 
        Rebecca Skye Dietrich (US enquiries) 
        +1 857 241 0795 
        prosensa@collegehill.com
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SRT-149 Myotonic Dystrophy & Autism Drug Candidate

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Good news today! Another company has launched a potential drug for myotonic dystrophy and by extension this may also treat the childhood forms of myotonic dystrophy. The childhood forms of myotonic dystrophy are highly associated with autism spectrum disorder, so it is hopeful that this new drug will have some effects on this as well as the cognitive effects in the adult forms of the disease. For the general autism population reversing the effect in the childhood forms of myotonic dystrophy. May help narrow the mechanism of action and suggest certain treatments in the future. The childhood form of myotonic dystrophy is one of the few single gene causes of autism. Because the drug will work through an RNA mechanism, it is  unlikely this drug will have a direct effect on the general autism population.

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Biogen / Isis Partnership Readies Autism Drug For Human Clinical Trials

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A not well know secondary effect of a drug that Biogen and Isis are jointly developing  may reverse some or all the effects of a specific single gene  type of autism.  This drug will be entering Human Trials in 2014 if all goes well.   In August Biogen and Isis announced a collaboration on a drug that looks extremely promising to treat a form of muscular dystrophy, Myotonic Dystrophy. But it may also reverse the symptoms of autism that occurs in some not well known forms of myotonic dystrophy in children. This could be a sleeper drug that may help with this larger and more pervasive societal problem.

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