At the Symposium, I asked the audience to write down what they liked about Symposium '98, and to share what they learned while attending the Symposium. Each family and professional received a questionnaire to complete. I said I would use this information in a review in the fall/winter issue of the Newsletter. I received responses from 30 family members and five professionals. Following is a summary of these responses.
"Did you enjoy the Symposium?"
Yes, yes, yes! No one marked "no" in answer to this question. Some even emphasized their "yes." They would all consider attending another one. Motel accommodations were rated excellent on 11 questionnaires; other ratings included: very good, superb, very nice, and good. The comments on accommodations included some advice:
- Excellent facility although a smaller facility makes it easier to interact with each other.
- Excellent, gets better with every Symposium.
- They were very nice, though the lecture room was often cold.
- Very well organized.
- It was wonderful, but as Ivan said during the Symposium, "I'd attend in a tent to be with these families and hear the latest."
- Very nice, but a little over-priced.
- Fine; there was plenty of room, and I didn't get lost going from place to place.
- Very nice - I think you should only offer the discount to parents who need it, and allow parents who can pay the full fare to do so. It is too much money to raise, and many families could have paid the extra two hundred or so dollars. (The fund-raising by Pennsylvania families provided enough money to pay a percentage of the hotel bill for each of the attending MSUD-affected families.)
This is how families answered the question: What did you like best about the Symposium '98 experience?
- Sharing with parents our experiences and learning from doctors and also parents.
- Substance-there was a lot of medical information.
- That I didn't have to do any work this time. I liked all the speakers. (from Reneé Eck who helped with Symposium '96)
- As always, the interaction with the families can't be beat.
- Everything nice and complete. Day was not long enough to get all topics in.
- It was great to hear from many different "experts" who work with people who have MSUD. The idea of parents and professionals sharing information is helpful. I enjoyed seeing the children with MSUD and how well many are doing. Also keeping up with families that have moved away and talking with professionals.
- Accommodations, good food, good organization, and amount of information given.
- I liked renewing old friendships, learning more dietary necessities - comparisons of leucine, isoleucine and valine quantities.
- Learning from Dr. Holmes Morton.
- Doctors and professionals' views and discussions.
- Talking, sharing with other parents and just being here and part of the big family.
- Everything was special! I liked having activities for children while we were in the conference room.
- The sense of a big family and the children all eating the same; comparing notes with other parents and seeing the kids growing up. Everything was great. I hope two years from today it goes just as well.
- Meeting new families! New information.
- Gene therapy update; leucine and isoleucine relationship and supplements; visiting with old friends and meeting new families.
- The discussion in the evening with the parents. Also Dr. Morton's speeches.
- It is always a highlight seeing and visiting with other MSUD families. Just the gathering is a blessing.
- Information! Networking; sharing experiences; catching up with families and seeing growth and development among the MSUD kids; socialization for Elan with other MSUD teens.
- Information; hearing accomplishments of research; meeting with other families.
- 1) The kindness and respect (love) that was prevalent. 2) The bringing together of, and the opportunity to befriend, people from all different cultures.
- That the information I'm taking back may improve my daughter's health.
- The baby sitters were wonderful!! I didn't need to worry.
- Can't say which - it was just GREAT!
What the professionals liked best:
- Just being with the families, learning their experiences and seeing the progress of their children.
- The genetic and medical information was extremely well presented, informative and timely. I enjoyed the biochemistry explanations.
- 1) The health care providers sessions, especially Wednesday at the Clinic. 2)Meeting new patients/families.
- Dr. Morton's talk on how MSUD affects the brain; seeing adults and children with MSUD. The meeting was well run and organized. I learned quite a bit.
- Sharing experiences with families. Very well paced. Excellent job of keeping people on schedule.
When asked if there was something they did not like about the Symposium, most answered no, or nothing, and some left a blank space. There were a few comments and suggestions:
- Everything was interesting; I think that the genetic research and gene therapy was a little confusing.
- We would have liked more small groups of parents with children of same age. We found the doctors meeting the most valuable.
- Some talks were more technical than they needed to be.
- Not enough time to talk in groups about common MSUD problems.
- We did not have enough time to get into small groups for discussion. More Dr. Buist - he's very entertaining!
- Too many scheduled meetings and speakers; too short a lunch break.
- Not enough time for all the questions, but could easily understand with all the people attending.
- Never seems to be enough time to cover all questions.
- Not all the questions on the index cards were answered.
- Excellent speakers, but some needed to put lectures on level we parents could understand.
- Gene therapy talk was over my head; it's so exciting and I wanted to better grasp it.
- It is hard to sit all day and into the evening. I would recommend 1, hours be scheduled into the program for exercise.
- It was difficult to keep comfortable, warm temperatures.
The parts of the program families enjoyed most and the reasons they were special:
In answer to these two questions, the three most frequently mentioned parts of the program were the three that most directly involved the families: the Coping Workshops, the Personal Perspective Panel, and the Question and Answer Panels.
Families could choose to attend a workshop for parents of teens, one for parents of children between the ages of 6 to 12 years, or one for parents of newborns to 6 years old. These sessions proved to be very emotional and extremely interesting and helpful to the parents. Here are some comments on the Coping Workshops:
- I enjoyed everything, but I found very stimulating the Coping Workshops - seeing all that parents are going thru and understanding what you are going thru. You can really realize that you are not alone and your feelings are normal.
- Sharing personal thoughts and ideas about MSUD.
- The families discussing their personal experience with their child or children was interesting.
- I liked the splitting of the big group, for a part of an afternoon, into several smaller groups. People are more apt to share their experiences when the group isn't so large.
Twice during the program, a panel of doctors fielded questions from the audience. The questions were written on 3x5 cards and handed to the moderators who recorded them on large flip sheets. Each flip of the paper revealed another question. This kept the answering very orderly. Following are comments:
- Learned day-to-day coping skills.
- The parents got a chance to ask questions; we need the professionals.
- Real concerns were answered by many different people to get more than one view.
- Good communication between parents and doctors.
- Everyone had a chance to ask just about anything.
- We picked up some useful information to take home with us on daily management.
Personal Perspective Panel:
On this panel were adults with MSUD who gave their views on questions asked by moderator Bobbi Wanta, and also answered questions from the audience. Many persons mentioned this as being particularly interesting. But the only specific comments were: that it was very interesting as far as coping with diet and peer pressure; and that it was very encouraging.
Dr. Morton's speeches:
Dr. Morton made sense when he explained the many aspects of MSUD that tend to puzzle parents. His knowledge of MSUD and personal interest in each child were evident and appreciated.
- Dr. Morton's explanations: It is encouraging to know that he is trying to comprehend the complexity of this disease, so that all of our children can benefit.
- Dr. Morton's presentations: His responses to questions were in layman's terms; very knowledgeable about MSUD. Also appreciate his research findings.
- I've been wanting to meet/hear him speak about MSUD since I had my daughter (3 yrs.).
- The amount of knowledge that Dr. Morton has and passed on to us parents. Especially the MSUD effects on the brain.
- I am really glad Dr. Morton wrote all his information down, so maybe our doctors will get a better idea on how to treat the children they see with MSUD.
- He was so easy to understand and everything made so much sense. Gave me some direction to consider to improve the treatment of my son.
Families found the information presented on the topic of Kathryn Burkholder's liver transplant of great interest; most stating that it was interesting and educational.
The possibility of gene therapy aroused a great deal of hope and fascinating interest. Some specific comments were:
- I enjoyed learning about genes.
- That MSUD may have a cure soon - I felt like it would not happen in my child's life time.
- Dr. Blaese's genetic discussion: it's hard to beat the kind of hope he's given that some day, in the not too distant future, we may have a cure for MSUD (or MSUD).
- Explanation of liver transplant and gene therapy gave us hope for the future
- Obviously the highlight and surprise was the announcement of a possible cure. Dr. Blaese probably never should have gone so far out on a limb, but now, I hope you can follow up and keep us informed.
- The session on gene therapy was very difficult to understand, but feel that the research being done is very hopeful for the future.
Comments from professionals about the parts of the program they enjoyed and why they found them interesting:
- The professionals' day at the Clinic on Wednesday was a unique experience.
- I only attended Friday but enjoyed the entire day. The speakers were interesting and interested in the MSUD young people themselves.
- 1) I enjoyed the health professionals session at the Clinic most. I enjoyed hearing about Dr. Morton's unique experience. He has what many of us dream of, the ability to get "STAT" labs and MRI's - which leads to insights and documentation of objective and quantitative changes needed to improve our understanding and ability to treat. 2) Also enjoyed visiting and "interpreting" for our patients and families. Our patients are always special.
- Arguments for better treatment.
How could the Symposium be improved?
Two persons felt the Symposium couldn't be improved. Others had some comments, ideas and suggestions:
- You all did a great job and the children enjoyed their workshops, which is important, too.
- Considering how many issues there were, I don't think it could have been much better.
- I wish it was closer. But then all the great people who made it possible probably would be too far away to attend! And it wouldn't even come close to this year's symposium. I've never met so many nice people in one place. How can we be kept up-to-date on the research?
- We need organized activities in the evenings so that families can interact.
- It would be good for parents to introduce themselves and give the name of their child when they speak from the audience. It would help to get to know the different families better. Possibly have introduction of families during a separate session - maybe the first night, so families can learn to know each other sooner.
- I think it would be good to allow parents with children similar in age to talk together for 2 to 3 hours. I think every group would then benefit from a "round table" discussion with parents from the next age group older to theirs. For example, parents with new babies might enjoy talking to parents of children 6 to 8. And parents of pre-teens would enjoy talking to parents with teens. What about more business time - not to talk about finances or anything else like that, but maybe things we feel the general support group should vote on. Or do we just leave all decisions to the board? (For example, name change from MSUD to MSUD)?
- One doctor was talking in terms I could not understand, and took it for granted that we all understood him. He was very hard to follow.
- I would be very interested in having the children with MSUD in a panel to express their feelings about their disease, like the young adults with MSUD did. Have them divided into different ages, so the parents can have a better idea of what to do better, or what to expect from the children as they grow.
- It would be helpful if the doctors could present their information in a more simplified form for ease of understanding by families who don't have a medical background. Some speakers did a better job with this, such as Dr. Buist, Dr. Kaplan, Dr. Mazariegos, and Dr. Blaese. I'd like to hear more about the day-to-day management of MSUD, such as how families do the diet, count milligrams of leucine, or count grams of protein? Do they keep daily diet records? Are low protein foods used? What is frequency of blood tests? Is urine testing routinely done and how often?
- Allocate more time for parent-to-parent interaction.
- It would be great if we could add a day and spend more time in smaller groups discussing our personal questions and problems. It would be great to have a list of all the people/places who are in attendance. This way we could seek friends and make new acquaintances of those in our areas. Unless you have met them before, or are introduced, you may not get a chance to meet.
- Need more than one hour for the break-out groups. Also, a medical professional could be added to each group.
- Group time for workshops increased to 1, or 2 hours; have a 3-day symposium vs. a 2-day symposium.
- Emphasis on managing day-to-day problems
- More professional interaction with patients and educate in the newer aspects of treatment and theory.
Suggestions for improvement from physicians and dietitians:
- More "doctors' panels" to answer patient questions from the audience - it is helpful for patients to have their doctor up there with other doctors treating the disorder and get everyone's opinion on issues.
- More recipe sharing - maybe a cooking class for parents and teens with advice on how to improve your own cooking.
- Sessions combining patients, their parents and professionals all together. When I am in professional and adult meetings, I feel that I am missing what the kids or their parents are saying.
- A workshop/talk on diet to see if we all manage it the same, or what other methods are used. A cooking class for parents to exchange ideas/tips/recipes and to help new parents overcome the fear of working with the low protein products.
Persons were encouraged to write more about what they learned from the speakers and in what way the Symposium helped them and their families:
- We attended the MSUD Symposium in Lancaster and want to extend our heartfelt thanks to all of those involved in the planning and implementation of the meeting. In addition to the meeting itself, the level of support from the community was also impressive. Unfortunately, we were only able to attend on Friday, and as we were traveling with our 3 toddlers, did not have much opportunity for conversation with other families. Hopefully we will be able to remedy this at the next Symposium, which we will look forward to. - Karen & Jerry Dolins
- Obviously, we feel this way because Nikolai was on the panel, but it was touching to hear from the adult MSUD for the sake of the younger ones. It probably helped the parents. I'm sorry not more younger children attended.
I think the location was the key to having so many attend. If Boston is chosen next time, there may be interest in new speakers - only if Dr. Shih takes an active role. But it will be much more expensive. Nevertheless, no future symposium can be planned now without specific attention given to the progress of the cure. It may be necessary to direct the location around that company.
I think all parents should take a moment to consider how difficult it is to have 400 people, with kids who have special needs, come to talk, eat, sleep, play, and learn. We are so busy, even this evaluation is late. Our hats are off to the organizers! Barbara and I think they did a fabulous job, down to the details of having gifts in each room!
The only other issue which may be raised is the silent one of how the doctors disagree with one another. It can be difficult for parents to be convinced of one approach in a lecture, recommend it to their doctor, only to be told that it is a wrong, or at least not necessarily the best, approach. That is the conflict of getting parents involved, but as you know, it is needed.
Nikolai's comments: He liked it. The only negative: kids didn't have much time to get to know each other again, because they kept being separated. Everything else is positive. - Eric & Barbara Rudd
- The information I have learned at the Symposium will be brought back to my son's metabolic physician to update her on new findings and research. - Jody Carrington
- I always enjoy attending the Symposium even if I don't leave with more information. Just the discussions with other MSUD parents makes it worth attending. My problems don't seem so bad after I talk with other parents. It is nice to just visit with everyone. It also gives my MSUD child an opportunity to be around other MSUD children, and she doesn't feel like she is the only one with the disease. - Mary Ann Peters
- I have been reading and re-reading the manual that we received at the Symposium. There is a wealth of information that can benefit all of our MSUD children. At the Symposium, I learned a great deal from Dr. Morton including the relationships between leucine, isoleucine, and valine and other amino acids. Also, the effect on the brain and development of MSUD children when these amino acids are not balanced correctly. It was encouraging to learn that Dr. Morton has gained an understanding of cerebral edema in MSUD children.
Dr. Naylor from PA spoke about the benefits of mass tandem spectrometry for newborn screening. Wouldn't it be wonderful in the future if all newborns could be screened so quickly for so many rare diseases? Dr. Michael Blaise from the NIH explained about chimeroplasty. It seems to be the most promising gene therapy for our kids. It is encouraging to know that someone is working to improve our children's future and that the liver will likely be the targeted organ - Sandy Bulcher
- Maybe we don't need another MSUD Symposium in two years if these children can all be healed!J We appreciate the good clinic and care we get in this area after hearing of the care some others are getting. Really appreciated that Neil Buist could be here and sure enjoyed his talk. We need more people like him. Very thankful for what Dr. Blaese is doing and sure hope it will work out. - A parent
- I had a great time and my son too. Even with the long drive ahead, I would do it every year. I can see there is a lot of hard work and time put into it. God bless everyone who did this Symposium. We really enjoyed this time we had together. See you in two years in the year 2,000. - Lucy Silva
- This one child threw us into a frightening world. But now it is a big part of our life, and we love it. We are happy to be a part of this family. Life would seem so empty without it. We made more friends through this one child than through all our others combined. This includes not just the families, but the professionals as well. Our love to all. - Ivan & Katie Fox
- After her liver transplant, Kathryn Burkholder asked her mother why she was never 6 or 7 years old. "I was 4 and 5, then 8 and 9, but I wasn't 6 or 7." (The years she was very sick.) "Other children are 6 and 7 years old but I wasn't." - Kathryn's grandmother, Mrs. Isaac (Rachel) Newswanger
- My nephew (who has MSUD) enjoyed the socializing. For me (great aunt, aunt, grandmother, and mother), it was wonderful talking to parents of children the same age as my great nephew, finding similarities of problems, hearing other doctors, of course, especially Dr. Morton. All were wonderful. Dr. Blaese (God willing the cure) and Dr. Kelly and Dr. Cox so brilliantly explaining the chain reaction of trouble cells. Thank you. - Sheila Wasserback
- I feel grateful for Dr. Morton's help for our problems over the years, and for the doctors that traveled from Philadelphia, Pittsburgh and many other states to help with the "forward movement" of new information to help or cure MSUD patients. Thanks so much to all who made the Symposium possible. - Norman & Mabel Burkholder
- We learned that we have much to be thankful for to have Dr. Morton as our doctor. Talking with some other parents you find out some doctors are still doctoring the way they did 15 to 20 years ago. We also found it very interesting to see all the adult MSUD doing so well. - unsigned
- I have benefitted greatly from this Symposium and will now take back ideas on how to better care for my daughter, Samantha. Her life will be better because of this.
Thank you for making it so well organized. Every minute was utilized to the fullest to bring us the most information possible in two days. Thank you for the nice gift basket. It was a nice touch to the end of a hectic, long journey.
I admire and appreciate the strength and comradery of this community and wish I lived closer. We are looking forward to the next Symposium. A million thanks. - Barnfather family
- The biochemistry was so interesting! I only deal with MSUD on a personal basis and to delve into the disorder itself was very educational and fascinating. - Patti Lee, CNM
- I am a health care provider, so my perspective is a bit different from a parent's. I am responsible for a unit at the state health department in Maryland that works with 8 children (at the moment) with MSUD. Our hopes for the program were to learn about new ideas in the treatment and diagnosis (through newborn screening) that might help us better care for our MSUD patients and to find them more quickly
The unique experience of Dr. Holmes Morton (with a large number of very compliant patients, from a very similar genetic background, living in a relatively small area, allowing him to follow them very closely) has produced a number of insights that we hope will help us to manage our patients better. We are pursuing a series of strategies to speed up our newborn screening test turn-around time and some insights into doing this were also gained.
Specific efforts will be made to implement DNPH monitoring, to set up full amino acid profiles from fingerstick dried blood spots with HPLC and isoleucine/valine supplementation. (Na balance measures are already in more general use.) We also would like to try the sick day regime. But implementing this on a population basis in a state system will require educational efforts with our metabolic specialists, working together with our families to do the therapeutic trials, and scraping up a few dollars for the improved lab capability.
Immediate major efforts in newborn screening will be focused on improving transit time from the hospital (where a specimen is drawn on day two) to lab for testing. Tandem mass spectrometry is a longer term goal. - Susan Panny, MD
- I liked the way questions were handled when there was not enough time to address them after the particular speech. The flip chart was a great idea! - A professional
- I don't think I got across the idea that I wanted to, namely that the powdered beverages are an excellent source of total nutrition, albeit providing an inappropriate protein/calorie balance for older children and adults. We need to devise ways of giving them either a flexible source of more protein (i.e., amino acids), or calories, and/or vitamins and trace elements. Anything that is manufactured with this in mind to treat PKU, MSUD, etc. could be classified as a medical food. - Neal Buist, MD
The Symposium Committee worked very hard and did a splendid job. They had good support from many other families in Pennsylvania who were all asked to help with fund-raising. They raised well over $30,000 mostly through individual donations of a few dollars to $1000, except for $10,000 given by the families in Blair County from the proceeds of a fund-raiser auction. Families and their relatives prepared low protein foods, helped with child care, planned the teen program and entertainment for the children, provided for transportation needs and other details. A united effort paid off in a truly successful symposium.
Low protein foods, formula displays, food supplies and monetary contributions came from Applied Nutrition Corp., Dietary Specialities, Inc., Ener-G Foods, Inc., SHS North America, and Ross Products Division. Their contributions and support are so needed and appreciated.
United Service Foundation, Inc. provided $12,500 specifically to help families attend who had never been to an MSUD Symposium before, but were now able to attend with some financial assistance. These families genuinely appreciated this opportunity. Our sincere thanks to the Weaver family who administers this foundation.
The committee greatly appreciated the wonderful cooperation of the staff at the Willow Valley Resort where Symposium '98 was held. They were willing to do everything possible to accommodate our MSUD group. They graciously worked with the committee in planning the menus around foods appropriate for those with MSUD, even making the low protein pastas. A hearty thank you to the staff at Willow Valley Resort.