I recently became aware of a new and exciting company working on a potential drug therapy in the Myotonic Dystrophy space. Valentia Biopharma S.L. is a biotechnology company born in August 2006 as a University of Valencia spin-off.
NIH officials announced yesterday that a new contract has been let with UCLA to form a network of researchers at various academic institutions to identify promising new and older drug compounds to treat Autism Spectrum Disorder (ASD) and to see if they merit additional investments.
The program is part of a new initiative called “Fast Fail” intends to vastly speed up the drug development process and reduce the costs of this drug development. Instead of taking years of work to see if a drug works this Fast Fail process could see results within weeks. As ASD is a huge issue for the USa and other countries this fast testing with myotonic dystrophy drugs could lead to treatments in a much faster time frame.
Dr. James McCraken who is leading the effort at UCLA states “The Whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe and thereby speeding the development of effective new therapies and reducing the overall cost”
A highly promising Compound has formed the basis for a new pharmaceutical company. This company is Called Atricode and is based on compounds that have been researched at University of Southern California to treat and cure Myotonic Dystrophy. Sita Reddy’s Lab has been instrumental in moving these potential treatments (MDI16) for Myotonic Dystrophy. The lab and company has recently been awarded a $90,000 grant through a competitive process. More about the grant:
2012 USC Ideas Empowered Program Grant: $90,000
Atricode is developing treatments for rare diseases. The lead indication, Myotonic Dystrophy Type 1 (DM1,) is a devastating genetic multisystem disorder with no available treatment or cure. This team identified highly potent and selective small molecule leads that rescue DM1 pathology in patient myoblasts and in DM1 mouse models. The team joined forces with experienced entrepreneurs to form a start-up company that moves the drug candidates towards the clinic. The drug candidate could be the first therapy to treat this devastating disorder and the Ideas Empowered funds are critical for the selection of the lead candidate for clinical development and to support fundraising efforts.
The Muscular Dystrophy Association of the USA (MDA) announced its winter research grants. 13.6 million was awarded including two grants for Myotonic Dystrophy research. One grant to doctor Disney will explore the possible therapeutic effects of small molecule approach for DM2. This would be a strong complement to the DM1 research that is ongoing in his lab.
The other grant to Dr. Mockton is to research the effect of Triplet expansion and stability on the likely course of the Myotonic Dystrophy Disease.
MDA is spending 4.6 of its research grant budget this winter on Myotonic Dystrophy Research.
My son Chris has had multiple issues with Pneumonia including multiple hospitalizations and ventilator support. I found this nice article on Respiratory support that is pretty technical. The part on General measures is below and you can link here to the actual article..Respiratory Consequences or Neuromuscular Disease. We have done the following with Chris
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