The Pringles: SMA Conference in San Jose, CA

June 24-28th we attended our second SMA conference. This time we headed to sunny California.

This year we had Gayla, Bill, and Yvonne (Maya’s nanny) come with us to the conference. The first day they had classes for newly diagnosed families. Even though we are a year and half in, they still considered us new. That night, there was a nice reception dinner for just our group. It was sad to see so many new faces from last year. A few of the families we recognized, but amazing how many more there were – from all around the world, too. There were people there from India, Africa, China, etc. This SMA conference is the only one of its kind so everyone wants to come and hear what all the doctors and therapists have to say.

Friday we started our classes and Maya went to the child care. She had so much fun and some of the girls and boys from last year recognized her and they were just called her name from across the way. It was so cute. One of Maya’s favorite buddies was also there – Billy. He is a black lab from where I can’t remember but the family he is with has a boy and a girl with SMA. She was so “cited” as she says to see Billy. Some of the classes were the same as last year, but since we know so much more being a year later, it was very helpful to know that we have the right equipment, all the types of doctors on her team and all the therapists. It was a refresher, but also a much better chance to interact with the other parents. In particular was the Type II class that parents go to. We learned so much about potty training and preschool. I think we took 3 pages of notes and caught up with about 10 different couples. Friday night was a banquet for everyone. Maya made a new friend at the table (her brother has SMA and could not fly from NY). Charlie and I were called up during one of the award presentations for the fundraising we did for Unite for the Cure. It was nice to be recognized like that.

Saturday was just more classes and going out to eat at places like In and Out burger (which by the way became quite the favorite place for B&G). We put Maya in the power stander, Standing Dani, again and this time she just took off. Last year, she was just learning what to do but this year, I think she was going around in it for about an hour. I would tell her to come back and she would just keep roaming the halls. She definitely liked it and cried when we took her out. The only problem about getting this is the cost. It is about $16k! Insurance is highly unlikely to pay for it since they are paying for a manual stander, a manual wheelchair and a powerchair. But standing is so important to these kiddos and there are so many benefits that some kids at her age don’t even opt for powerchairs! They sit at times, but their primary mode of mobility is that Standing Dani! Since the powerchair is about the price of a Honda Civic we opted to get that through insurance and then try to save up enough to get Maya the Standing Dani hopefully next year. Charlie and Maya got in a little pooltime as well…

On Sunday we had a chance to listen to the scientists and pharma companies who are currently doing research and developing drugs and stem/gene therapy treatments for SMA. It seems like there are several great leads out there. The one we helped fund is about to present their final case to the FDA for stem cells. If we are lucky, the clinical trials on Type 1 patients can start right after the FDA approves – they have 30 days to review the information but often put things on hold for safety questions – they are thinking later this fall or January. The first trial will last about 12 months. If Maya were ever to be considered for a trial, it probably wouldn’t be until Phase 3 when they accept a broader range of cases. This could be another 5 years from now but we still keep the hope. Another promising treatment is gene therapy. There is a company that is literally injecting the missing SMN1 gene into SMA mice and monkeys. There are seeing exceptional results. Other companies are working on conventional drugs like Maya is taking now. I think we heard about 7-8 programs going on and they say that only 10% make it to FDA approval. That being said, we are definitely eager to have a treatment sooner for SMA, but just looking in the past 10 years, they have gone from 1-2 possibilities to 7-8. That means that more scientists, universities and pharma companies are paying attention to SMA and with that we have more hope.

Sunday afternoon we headed to San Francisco for the day. We went to Fisherman’s Wharf and saw the sea lions and Golden Gate bridge for some pics. We dropped Bill and Gayla off for their continued vacation for the rest of the week and headed back to pack up and get home on Monday. Fun trip. We can’t until next year when the SMA conference will be at Disney World!