I've heard that the UW has much better care for the boys than Children's - that basically, the whole care system is set up to focus on pallative measures, so once we're at that point, I'm sure we'll be taken care of.
The thing is - the paradigm of duchenne treatment is changing. It's no longer simply pallative, but strategic - how do we treat these boys now with an eye toward any treatment that might be available in the future so that they can take advantage of it. And if Children's isn't looking in that direction, but they're taking MDA money to be an MDA clinic, then they are doing a disservice to every patient that might be able to benefit from future treatments that they serve.
The people I met with actually asked me to meet with Dr. Hays to explain to him what Cincinnati does so that he could be a better doctor. They said that he really wants to be better, and isn't that important, that he wants to change? I said, OK great, but when I want to get better at something, I get off my a**, and figure out how to get better at it. I don't sit around and say, "Gee, I really want to be better at this. I wish someone would come and tell me exactly what I can do to get better. And until then, I guess I'll just sit around and wait..." I mean, seriously, I'm supposed to sit down with him and tell him how to be a better DMD doctor? What I want is someone like Dr. Wong, who actually cares enough to have the intrinsic motivation to be the best doctor she can be to these boys.
More venting. Sorry...
I really have no comment because I'm not sure what it means for the families but thought I would post it.
Muscular Dystrophy Association Launches New Venture Philanthropy
Will Apply VC Business Practices to Funding Research
TUCSON, Ariz., April 1 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) today announced the launch of a new initiative, MDA Venture Philanthropy (MVP), which will be a wholly owned subsidiary of MDA. A 501(c)(3) nonprofit organization, MVP will break with traditional philanthropy models for funding research by applying the tools of venture capital firms to fast track the development of new therapies and cures for muscular dystrophy and related diseases.
In the newly emerging realm of venture philanthropy, MVP will be uniquely positioned because of its ability to build upon MDA's highly successful translational research program that efficiently integrates the various steps in research and clinical trials, while leveraging MDA's significant clinical resources.
MDA will provide initial seed money of $10 million for MVP's operating capital. Its investments will specifically focus on companies conducting commercially promising research in four disease categories: Duchenne muscular dystrophy (DMD); amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease); spinal muscular atrophy (SMA); and Friedreich's ataxia, myotonic dystrophy, and limb-girdle muscular dystrophy.
MVP will approach the raising, investment and management of funds much like a venture capital firm, utilizing venture capital principles and tools to develop strategies, evaluate each investment opportunity, make quick and soundly based decisions and comprehensively report results against milestones to investors.
MVP will seek donations from a new body of philanthropists -- individuals with both a familial connection to muscle diseases and the financial ability to make a significant contribution ($250,000 or more). MVP will then use the donations to place initial targeted investments into biotechnology companies and academic institutions with highly promising therapies for these diseases. These investments by MVP will not only provide needed funding but will also serve to attract follow-on investments from other sources such as pharmaceutical and biotech companies.
"We're excited about MVP because it will help us to overcome the critical funding gap that can occur in research on promising new drugs and therapies," said MDA President & CEO Gerald C. Weinberg.
MVP's venture capital model addresses the well-known drug development challenge in which promising work on ideas for new drug therapies, development of which can take many years and cost more than $1 billion to bring to fruition, can run out of money and be abandoned. The funding problem is worsened when the drug or the therapy is being developed for a disease that affects a relatively small patient population base, with a relatively small return on investment. MVP overcomes these problems by joining the entrepreneurial spirit of donors with best business practices to drive a specific therapeutic development.
"As a venture philanthropy operating in a very distressed economy and a competitive philanthropy landscape, MVP will hold the companies we invest our donations in fully accountable through milestone-driven contracts," MVP Executive Director Sharon Hesterlee, Ph.D. said. "But we will also hold ourselves accountable to our donors through a structure that enables them to have an advisory role in MVP's investment evaluation process, and to see firsthand that MVP is meeting its goals as an organization."
About MDA: MDA is a voluntary health agency supporting programs of worldwide research, comprehensive services, advocacy, and professional and public health education for muscular dystrophy and related diseases. MDA is the largest nongovernmental sponsor of neuromuscular disease research. The Association's programs are funded almost entirely by individual private contributors. Visit www.mda.org for more information.
Contact: Tara Wood
Director of Communications
Wow, this is exactlly how I feel. I love the MDA and I really want to be supportive of the MDA. With that said, its maddening that they have raised so much money over the years, and the progress has been minimal. There has been more progress since the PPMD has come around than in all the years the MDA has had there fundrasing. I think their intentions are good, but possibly they could use better management. I feel though with the amount of money they have raised, there should be more knowldege and help out there. Its got to where you only see a rep from the MDA when its fundrasing time.