Hello,
I just met with three members of the MDA, one of whom heard me mention at a Darius Goes West screening that we hosted that I was not happy with how aggressively they funded research.

They wanted to talk with me about why I take Aidan to Cincinnati instead of going to Children's hospital in seattle, (where the neurologist told another family that prednisone doesn't stunt growth like deflazacort!). They also wanted to talk about what they could do to earn my support.

They brought what they thought were "supportive documents". They included a list of all projects funded by the MDA for the past three years. It included a $34,000 grant to Dr. Hoffman for non-hormonal steroids. They also brought an MDA press release about Dr. Hoffman's recent results in the puppies, where they "announce" that the "MDA-supported investigators" had a recent breakthrough in exon skipping.

I left the meeting in tears. I feel that the language of that press release is very misleading in that the MDA is basically trying to get people to think that they are funding Dr. Hoffman's exon skipping efforts, when they haven't spent a dime on exon skipping. They also recently just opened a new office in my home town, like the one in seattle wasn't big enough for them, and I'm sure they spend at least a couple of hundred thousand per year on it. And yet Dr. Hoffman is only worth 34K to them? I really hate to think about what could have been accomplished by now if the MDA was serious about actually curing this disease rather than loaning my son some crutches.

I'm sorry about the tirade - I just got so frustrated with them asking me, "How can we serve you better?", and I kept saying - take some of that 162 million you have in the bank and spend a couple million on research that actually might hit the clinic sometime soon rather than spending a pittance on a bunch of projects so that they can take credit for their success. Only a handful of grants they handed out were over $125,000.

At this rate, they'll cure duchenne in 50 years. At least they'll all have jobs until then.

I'm really sorry if I offended anyone - just been carrying this around since yesterday.

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Hi again

Vent away. I understand. When we were looking for care, Dr. Wong's clinic was just getting started. I met her at a PPMD conference quite a few years ago. She was there as an observer, not a presenter. She is very passionate and focused. Dr. Hays sees MDA clients 4 days a month. He has other clinics on other days. I haven't experienced a leadership role from him. I bring him things that I want to do. He asks why, then we do them. He is much more of a responder than a leader. I don't see him instigating a strategic model. I just wanted you to know that the clinic does go beyond MDA and that relationships can get you what you want as long as you have a way of knowing what that is. I think you are right about a change in the care of dmd. I am so glad it is happening.
Karen

Mindy said:
Hi Karen,
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...
Thanks, Karen. That is good to know. I think I'm just freaked out enough about the whole thing that I'm tired of being the expert. I need someone right now that knows more than I do about duchenne as Aidan's doctor - it's just too hard to always be on top of everything.

And now I've got to battle Dr. Valerie Cwik on top of that...
Firstly let me say that although I am just about to offer a differing opinion, I am by no means a fan of MDA (Australia) - in fact I have found some of their actions to be down right shameful. I do however understand WHY they have to afford the same priority to other orphan diseases and neuromuscular conditions that come under their umbrella as they give to DMD/BMD.

As a mother of a son with DMD, I want the whole world to stop focusing on cancer, diabetes and obesity and find at least an effective treatment that may buy us some precious time - all the other major diseases (even cancer) have treatments that can at least offer hope, or a means to manage the condition - and I think its high time we all got to try the "hope shirt" on and see how it fits.

As a realist I find myself recognising that if charities such as MDA do not retain a focus on conditions such as Motor Neurone disease, Congenital MD, Limb Girdle MD (to name just a few) there may be healthy children forced to deal with becoming carers before they're in high school - or even worse, children that have to deal with the loss of a parent, which I think is just as hideous as the fate we face (only we have the benefit of "maturity" to help us through the heartbreak and tough times). Which plight is worse? I don't think either "out ranks" the other, but I also don't think any of us would force a child to have to deal with the emotional turmoil we face, because we pushed for MDA to give our sons condition priority.

Add to this the fact there are so many parent based charities supporting solely DMD/BMD, and you have another issue that is fueling the fire. All the time DMD has multiple charities funding research, it is very hard for MDA to justify giving the lions share of research funding to DMD over their other conditions, which may not receive any additional research funding from other charities.

This being said I do not condone the "parading" of our children to raise awareness (aka sympathy) therefore opening the publics purse strings. However even this is not an issue that is controlled by MDA - it has occured as a sad result of human nature. If MDA were to utilise more adults in their telethons and mailouts, the amount of donations received would probably drop, as an adult with a neuromuscular condition is no where near as heartbreaking (and therefore worthy of Joe Publics hard earned money) as a child. If they were to practise open and transparent fundraising, the reality is the amount they received would probably decrease, and then DMD would receive less funding support from MDA, not more.

I do think in the current ecomonic climate charities should review their funding commitments - especially when it comes to paid staff verses volunteers. The sad part about a country in recession is that there are so many people out of work, and charities could be utilising this to their benefit by offering volunteer positions (that enable people to diversify their skills in the hopes of gaining paid employment when the situation improves). Every dollar saved on wages is another dollar for research, and I know there are a lot of unemployed people out there that would welcome the opportunity to work - even if it does mean as a volunteer - as it is a wonderful thing to have on your resume and is often well regarded by future employers.

While it may seem I am defending MDA and their misguided approach to our sons condition, I am merely trying to point out that MDA are really just another corporate enterprise, that will do anything they can to guarantee donations in an increasingly difficult financial situation. Its not fair, but we know better than anyone that life's not fair - otherwise our sons would be healthy and DMD would not even be a disease that was still waiting for a cure.

Is there an easy solution? I don't know, but I do know that myself and another mum took on MDA over here, and made it well known what our issues were. When our feedback fell on deaf ears, we took measures into our own hands and started fundraising to implement a quality of life programme for our boys. Once it was all up and running we "handed" it over to MDA (who of course took full credit for the initiative). Whilst that really hurt, the end result was we got what we wanted - even if we did have to watch while the general population applauded MDA for their insight and hard efforts.

Never forget that en masse we have the ability to bring about change. And that once we have had the opportunity to vent, we can take the major issues that affect families and work together to change things for the better. It is so lovely to know I'm not the only one that thinks we are living in a world gone crazy and if I could, I would hop on the next flight over to the USA and add my support to your efforts. :-)

As far as Dr Hays goes, speaking from a mum who lives in a country where we don't even have access to a neuromuscular clinic, (or regular physio, OT etc) I find myself envious of the families that have access to Dr Wong. I do however find myself wondering how Dr Wong feels carrying the hopes of a nation on her shoulders alone.

What a wonderful thing it would be if families worked with Dr Hays to help him emulate the services of Dr Wong, therefore allowing her ever growing workload to be shared with another equally capable doctor. I wonder how Dr Wong feels when she wants to take vacations knowing there is no-one that is able to help her patients out in her absence, or how much pressure she is under to go to work even when she is sick as there is no-one else to steer the ship? Whilst Dr Hays may seem slack and unmotivated, maybe he sits in awe of Dr Wong, and is looking for guidance from parents so he can be the kind of doctor they want him to be, rather than the kind of doctor he thinks families need? and maybe (just maybe) if a few dedicated families embarked on this journey with him, his client base would increase, and therefore he would be able to dedicate more than 4 days per month to DMD.

They say its lonely at the top, and I should think it is very lonely for Dr Wong as a pioneer in DMD care. What a great gift for her to be able to have a fellow doctor to run ideas past, and share information with or debate the efficacy and expected time frames of current research..... personally I would give my right arm to have access to a doctor that not only values a parents input (and considers them a productive member of their sons care team) but also encourages feedback to improve the service they provide.....but that is just me, and I do appreciate how as parents it is easy to feel like you have enough on your plate getting your family through this journey, without having to drag a doctor along for the ride.

Well thats my two cents worth (and another post that gives War and Peace a run for its money). Thankyou for giving me the opportunity to vent with you all, and I hope one day we will all look back on threads like this and say "can you believe things were ever that bad?"

Hugs to you all,

Jules
I just have to say that in regards to Dr. Hayes. There have been many many parents that have talked with him and nurses ect. I certainly would not give up my right arm for the care we recieved. I know another Mom that also talked to Dr. Hayes about actually talking with Dr. Wong many months ago. Again, that never happened. Mindy has met with another Neurologist in Seattle and explained what they need to be doing. Again, nothing happened. Not only that, but my cousin passed away 20 years ago from DMD. Has anything changed in Seattle? Only the fact of steroids which they recommed starting too late. It's time for a definate change in Seattle. I know Mindy and other parents that have been crying out to them for a change. I say so much for the Neuromuscular clinic here in Seattle, might as well not have a doctor at all. That is why Mindy and I spend the money to take our kids to a specialist that cares.
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
(520) 615-6700
twood@mdavp.org
www.mdavp.org

http://news.prnewswire.com/DisplayReleaseContent.aspx?ACCT=104&...
Wow! Thanks Donna for finding that article. mmm...But I wonder...how much is it gonna cost MDA to jump in the VC game? Sure sounds like additional administrative costs to me...could this mean LESS donations filtering into the lab?

Jacobs Mommom said:
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
(520) 615-6700
twood@mdavp.org
www.mdavp.org

http://news.prnewswire.com/DisplayReleaseContent.aspx?ACCT=104&...
MVP sounds somewhat like project catalyst that PPMD started 5-6 years ago? Sounds like a rip off to me. I can't gripe too much if they are successful, because that would mean more $$ for research.

Karen
Very well said Julie. While MDA has its fault(as does all organizations), they do cover many many diseases. Parading kids out is just the way to raise more money. EVERY organization will parade out kids for the sympathy factor. We split between two local mda offices. Both with their good and bad points. Our local doctor has almost NO bedside manner, but she is up to date on research and knows and communicates with Dr. Wong. She listens to us and we get what we need and want and the care we should be getting.

Oh, and our mda clinic DOES promote ppmd. They will tell you about it and when ppmd started the registery, they had fliers up telling you where to go and how to register and asked us if we had done it yet.

--Samantha
Hi Samantha,
I understand that the MDA covers many diseases. My main complaints about them are:
1. The deceptive nature of their press releases, leading people to think that they are supporting research that they indeed are not.
2. The incredible amount of money that they spend on themselves and their own infrastructure
3. The lack of urgency to find a cure (reflected in the huge amounts of little tiny grants that they give)
4. The lack of accountability the hold any of their official "MDA clinics" to. I think that if you put your name on a clinic and give it money, you should be sure that there is some sort of standard of care that is given there (ie: that the doctors who run it are familiar with one of the main side effects of the only drug they have to prescribe)

I'm sure I could come up with more, but up here in Seattle, the MDA seems more interested in self-preservation than they do in serving families.

Mindy
I know it's been a few months since this happened to you, but I'm sorry all the same. I can't imagine how you must felt. I appreciate all MDA does for people, but about a month after Wyatt's diagnosis (he was 11 months at the time) the MDA rep I met in Seattle gave me brochures about summer camp and wheelchairs. When I met Pat Furlong in April, she did just the opposite. She gave me HOPE. She talked to me about treatments, suppliments, studies, stretches, etc. I know that summer camp is a great amazing thing. I didn't want to talk about summer camp. I wanted to talk about how to help me baby. They (seattle) told us to take him home and love him, that's all that could be done. However, Pat introduces us to Dr. Wong and now Wyatt is getting what he deserves A CHANCE. Thanks for continuing to fight this disease for "our boys!"

Melissa
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.
What you've got to remember is that the MDA covers many more diseases than just Duchenne. They were also founded during a time when there was no hope for a cure in the forseeable future. So they chose to focus only part of their resources on medical research and much of it on social services. So while PPMD may be where our hopes and dreams lie for a real future for our sons, the MDA is really the best option for families with all dystrophies who are struggling with how they are going to afford the next piece of medical equipment.

The MDA is run largely by professional fundraisers and social service professionals, where PPMD is run by parents living in the trenches. So we naturally feel more of an affinity towards this group. They each have their place and are both worthy of our support, but we have to be realistic about what the MDA can really do for Duchenne.

MommaToo said:
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.

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