• · We have met most of you, but for those who don't, this is Jon and I am Rashmi. We are the parents of a four year-old child with two, actually three rare diseases (SJIA, ILD and PAP) and we are on a quest to get him better. Apart from being parents, we are also startup entrepreneurs and look at any problem with a startup perspective.
• · In the years since my son got sick, we have learnt so much
• · We have learnt how little is known about the disease my child has, why he got it, how to treat it. As many of you sitting at the table have told me in conversations, we don't know what this beast is.
• · We have learnt how busy doctors give so much of themselves to kids with rare diseases , trying to understand them, help them get better.
• · We have learnt about the secret, strange world of kids with rare diseases : desperate parents connect to learn from each other. We have met other kids like Vikram, who have the same two diseases, and now our fight is for them too.
• · We have learnt a little about how interesting system the immune system is, the checks and balances it has, the redundancy. Although I gave up science quite a few years ago, I can appreciate the beauty of the system. I don't know enough about the lungs to appreciate it yet, but maybe by the end of this conference.
• · Jon and I think of chasing our son's disease as chasing a train . We are chasing and trying to outrun it. We know the train is moving, but if we run fast enough, there is a chance to find out what this is, treat it the right way, get him better and live happily ever after
• · Ok I am realistic, the live happily ever after does not happen , but I would take slightly more normal life
• · We are running because we want to save our son's life , and that of the kids like him. We are running because we want to do it before my son realizes what a crappy deal he has in life. He already knows about the SJIA monster and how it lives within him and he has to take drugs to try to kill it, but he does not know that this is not normal. We want to run fast enough that we can get him better before he realizes how sick he is.
• · So what can someone like us do as parents ? Turns out there are models. We delved into the world of parent foundations, met with many parents of kids with rare diseases, many of who have left successful careers to try to find a cure.
• · In trying to understand what makes parent run foundations effective, we came back again and again to the lack of connections between the different groups . And that's where I feel like we can make a difference. One of my advisors during my postdoc, in writing about web revolution wrote about how networks become exponentially more valuable with more nodes getting connected. I think that's the case with rare diseases, so little is known about them that the more you bring people together, the more connections you create, you can dramatically increase the odds of finding a cure. What happens right now
• · Cases land up in individual doctors offices who treat them. Some get referred to specialists, most don't. For the interested scientist, it is so hard to find enough cases to find patterns . Betsy has worked so hard to find these cases of SJIA+ILD. Also HIPAA makes it very difficult to share cases without a lot of paperwork.
• · Parents are well connected with each other on Facebook. But that's mostly for conversation which is good, but it does not help us move research forward.
• · Scientists are connected at conferences, but its rare for them to gather like this and focus on rare diseases.
• · So if you look at this world, there are four to five players and connected within themselves but disconnected from each other.
• · So what are we doing
• · This conference and there will probably be others like it
• · A next gen drugs in SJIA conference in summer, bringing together scientists, parents, drug companies to discuss next gen medications
• · An online, patient-drive registry for SJIA, MAS, Stills Disease, NLRC4 mutation etc, so patients can self-register and agree to participate in research, and scientists such as yourself can have a chance to invite them to participate in studies.

In the meantime, the train we are chasing continues to move . My son's last sleep oximetry study showed a drop, he coughs sometimes now. Will we be successful? I don't know. But we have promised ourselves we will do everything to stop it.

Thanks for coming here on a weekend. Thanks to Robin, Fabrizio, all of you who have spent hours talking to me and encouraged me. Thanks especially to Betsy, for working tirelessly over months pulling this conference together.

We look forward to learning a lot this weekend.