Monitoring Ultra Rare (MPS/ML) Pharma

Posted Tuesday May 01, 2012 by Melissa

Prior to MPS coming into our lives, my job was as a strategic consultant, mostly to law firms. I sometimes joke that MPS still requires that I be a strategic consultant, my client is just shorter, faster, and infinitely more funny. But seriously, in the search for better treatments for our children, better environments at school, better and longer lives for them, we are all just that – strategic consultants.

And since we are all strategic consultants for the benefit of our children, one of our jobs is what is traditonally known as competitive intelligence. Now, in our case, its not really our competitors that we are concerned about, it is the group of pharma competitors that serve or will potentially serve our children in the form of treatments or cures.

So, let’s work to create a database of sources, academia, companies, established, biotech, early stage, rumored, etc. who are working on compounds that might be useful for any one or all of the MPS/ML conditions. Some are obvious, some are theoretical. But by collecting them and monitoring their activity, we can begin and continue our interaction with them, plan our strategies in terms of trials or any participation that we or our children might have with these companies, and analyze the different types of science that may potentially turn into longer and better lives for our children.

If you’re not sure whether a company would “fit”, include it and we’ll parse it all out later. If it’s a rumor, we can go with that for now because sometimes that’s where it all begins.

If you post companies below and what you know about their programs, research or therapies, I will continue the legwork on gathering information and make this database available to us all. Or, if you’re more comfortable, send me an e-mail or message using the contact page. I can’t do this without all of you, so even if it feels obvious (but please scour for the non-obvious), send it on!

What I’d like to compile in the end is something like this that a friend directed me to on the Parent Project Muscular Dystrophy page.

So start listing!

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3 Responses to “Monitoring Ultra Rare (MPS/ML) Pharma”

  1. Zacharon/Pfizer – MPS I, II, & III small molecule therapy for CNS involvement.

  2. PTC tHERAPEUTICS – Ataluran – Chaperone therapy
    MPS I – curently in clinical humsn studies or Duchenne Mucular Dystropy and Cyustic Fibosis.- Patietns must have atleat 1 non-sense mutaiton. – In skin biopsy phase in MPS I,

    Genzyme/BioMarin sponsor – NPS I Intrathecal clinicla trial for MPS I congition and MPS I-H clinical trial pre and post BMT.

    UofMn – Neuropsych Congitive long term study of memory and changes to function.

    MPS I Endocrine Growth Hormone study

    http://www.rarelydefined.blogspot.com

  3. Zystor / BioMarin

    Lysosomal company working on MPS III

    Kakkis EveryLife Foundation and Pharma Co
    kakkis.org

    Hide and Seek Foundation
    hideandseek.org