 Donna Appel is the expert, outside expert. She's hardly that. She's been to NIH. She'll be in this room or nearby the next three Mondays in a row giving her advice from the perspective of a nurse and a mom of a patient with a very rare disease. Donna? Thank you very much. I really appreciate being asked to come here. And I truly am a mom and don't feel like much of an expert personally. So I just want to give some thoughts. I in the context of our disorder, I really don't want to go through the description of our odyssey, but it's a life threatening disease. My daughter has HPS1. It's fatal and urgent. So we started without having an undiagnosed disease program two decades ago, being very isolated and very alone and tried to figure out what to do with this. And I laugh now. I kind of giggle about the new phrase, patient engagement and getting patients involved. And I kind of feel like, wow, we've been engaged for decades. And I think many, many families feel that way. I know certainly I do. Oh, sorry. But and Dr. Gaul's young pictures there with my daughter and our patient engagement began when I was supposed to collect 24 hour urine buckets from anybody I knew with the disease. And we had to do it in the wintertime because it had to be refrigerated on our deck. So patient engagement's been going on for quite some time. But gathering the patients, I think we're talking about common fundability. And the discussion this morning was about the high potential for making a difference in research. And I do believe that patients synergistically with researchers together can really impact research in many, you know, unique ways. Certainly, we started very small. And now we're in rare disease research network. And we just in February shook hands on the first clinical research center clinic for lung disease in Puerto Rico. So I just want to make a couple of comments that there's a fallout of access. So it's really exciting to get access to these brilliant minds in this network. But there's a little bit of a fallout from that because the families go through this odyssey and then get into this program and go, Oh, my God, I got into this program. That must be I mean, we're that's really bad that we're a person that can go into this program. So there's a bit of a fallout. And there's also the finding hope again, when diagnosed or not. So, you know, when you don't have the gene and you don't have the diagnosis, you have this dream in your head, like maybe it's not that bad, or maybe it won't be fatal. And then you get the diagnosis, and it's really great to get a diagnosis and you get that label. But then you have to find your hope again. So there is a certain psychological group of people that come out of programs like this that have unique feelings and unique things that have happened to them along the way, finding the home at the last chance hotel. That's kind of like what this is to a lot of us, like we have gone through this, trying to find a diagnosis. And this is the last chance hotel. But the fact that you're only there a week has nothing to do with the feeling in your heart of how close and how much you depend on the people that are at that hotel. So there's a great separation anxiety because those are the experts that kind of figured it out or certainly, even if it wasn't figured out, those were the experts in the country that were turned to by the local physicians that they depended on this group of people. So you as a mother then says that why wouldn't I want to live with all of you rather than where I live? So there is a great dependency even and not equal to the amount of time spent with you. Another issue is the research is not the primary care. I often worry about spending research dollars, you know, running my organization and having many people depend on researchers in the NIH. I try to teach my families, they're not your local physician, you cannot turn to them for your needs. And that has to be very, very explained. That has to be upfront and reiterated so many times for patients to actually be able to understand that really well. And then the primary care person suddenly doesn't feel that primary. There's a lot of co management discussions that we've had in this room today. And out in the world that sometimes doesn't work because the co managing local primary care person retires. And then certainly the next guy does not want to be that they just say, go to your experts. It happens over and over again that people are hospitalized and they're just told well, it must be a genetic thing. It must be part of your diagnosis. And we'll be like, it has nothing to do with the diagnosis. And they kind of just defer to your experts. So you get this kind of stigma world that you live in when you are so touched by such brilliant people. What can the UDN uniquely do? Well, in my opinion, and I don't know why anybody would care too much about it, but in my opinion, you are an example of a group of experts bringing together solutions to try to find a disease. Well, the same thing needs to happen for the patients and families that they probably would be benefit from being exposed to a group of experts on how to manage afterwards. So the transition of the discharge planning from the program should be that they are brought together by experts in the patient advocacy world. So not a bunch of, you know, a lot of times where in audiences where we have talking heads of great advocacy organizations that say that I did this and I did that and I did that. But if you look at advocacy organizations, you can tell what they're going to be good in. So some of them are going to be founded by IT people, and they have the great best social networking and platforms and apps to be able to run their disease. Other ones are founded by fathers who like might have been great and brilliant businessman. They have the best fundraising capability that you could ever see in your life. Some others are founded by mothers and they're so warm and fuzzy and like coping mechanisms and adorable. So in the in the fact that every disease has its experts and there are feels like neurology and cardiology. There are actually expert little categories of patient advocacy. So I would recommend that the mentorship program based from this should be exemplified by exactly how you're doing this. A patient should be referred to a bunch of experts that are uniquely good at one fundraising, one gathering a database, one giving coping mechanisms to the family, one, not just a single mentorship program, and then kind of allow the families to empower themselves to feel less isolated. And I think in that way, I am sure that Dr. Gaul in this program, not that I want to put them on the spot, but I'm sure that they there are a lot of phone calls that come back that that you have to the more and the longer and longer this program exists, the more and more you're going to be asked for follow up, follow up, follow up from the families because that's of course, we're smart enough to know where we want to get our answers from. So I think trying to empower them is the best thing to do. And I just want to say that, you know, Dr. Green was mentioning that it's kind of an analogous to the Wizard of Oz and it's very true because this program somewhere over the rainbow dreams come true. And that's what the families are believing of this.