Pfizer to Develop Families of SMA Quinazoline Drug Program

Yay for Pfizer!! They are right in our backyard! We are hopeful for their successful development for a potential treatment for SMA.

“Pfizer to Develop Families of SMA Quinazoline Drug Program.

Repligen Corporation announced January 3, 2013 that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s SMA program, originally in-licensed from 

Families of Spinal Muscular Atrophy (FSMA)



Families of SMA Conference at DISNEY WORLD!!

After almost a year of planning, research, and making reservations we finally made it to DISNEY WORLD!!  It was the trip of a lifetime and worth every second of planning.  It was every bit as magical as everyone says.  We were fortunate enough to have Grandma and “Aunt Ten” come with us and it was so helpful to have them there.  We were all so excited that I don’t think anybody slept well the night before our flight to Florida.  Once we boarded the plane we all sighed with relief that we were on our way!  Brooke and Brielle were great little travelers and Brooke even took a little nap in between playing games on the ipod.  Once we landed and made it to our hotel, we freshened up and then headed to the Spirit of Aloha Dinner Show for dinner.  It was a very entertaining luau with a traditional Polynesian band, hula dancing, traditional Samoan dances and the best part was a fire twirler!  At one point the cast asked kids to come up on stage to learn one of the dances.  They were asking Brielle to come up there on stage and she really wanted to go, so I went up there with her and helped her dance.  She had such a fun time and loved being in the spotlight!  After the amazing show was over, we started our walk home and looked up to see the castle in lights and fireworks above it.  It was a wonderful start to our vacation.

Day 2

“My Disney Girl’s Perfectly Princess Tea Party” at the Grand Floridian was beautifully executed to transform Brooke and Brielle into young Royals!  They participated in storytelling and songs and then Princess Aurora appeared from the back of the room and surprised all the little princesses!  It was the first princess they saw on our trip and she took Brielle right by the hand to sing a song.  There was plenty of time to visit with Sleeping Beauty and the hostess, Rose Petal, as they came around to every table so we could get autographs and hugs and kisses.  After that, they participated in a Princess Parade around the lobby of the Grand Floridian and had pictures taken on the second level of the stairway.  After this event, Sleeping Beauty was their favorite princess, hands down.  :)

“1900 Park Fare - Cinderella’s Happily Ever Dinner” was our first glance at CINDERELLA!! 

Cinderella, Prince Charming, Lady Tremaine (The Step-Mother), Anastasia and Drizella were all present and the girls could. not. wait. for them to visit our table!  We watch the movie, “Cinderella,” at least 3 times a day so they know the movie by heart.  We also have the movies “Cinderella 2 &3” and the girls watch them just as often.  It was so exciting for the girls to actually meet the characters.  Brielle brought her miniature dolls of all the characters and they were a big hit, especially with Anastasia and Drizella.  The Step-Sisters played for a long time with them, made up stories, and had very bad manners.  They were pushing and shoving each other and yelling across the room in very loud voices to their mother.  It was hilarious and they had 100% of Brooke and Brielle’s attention the entire time!  The Step-Mother liked Brooke and Brielle because they were so quiet.  She told them that children should be seen, not heard!  Their eyes were as big as saucers when she was around and they were on their best behavior.  It was so much fun to experience this through their eyes, they just could not believe they were seeing the real characters.

Day 3

The Bibbidi Bobbidi Boutique…….  ahhhhhh, what more can I say.  Mommy and Daddy were teary-eyed watching our princesses turn into sparkling princesses!  The Fairy Godmothers-In-Training gave them up-do’s, painted their nails and did their magical makeup.  It made my heart swell and I could have filled a 16GB memory card with pictures on just this, but I’ll spare you of that and give you some highlights.  :)

After getting all beautiful we walked across the hall of Cinderella’s Castle to have dinner at Cinderella’s Royal Table.  Brooke and Brielle were greeted by Cinderella and we took lots and lots of pictures.  They were fascinated with her long white gloves.  They were both given an “Honorary Princess of the Day” sticker and a plastic sparkling wishing star.  After that, we went up the winding staircase to the dining room and were seated at our table decorated with Princess confetti.  While we were eating, we were visited at our table by Ariel, Sleeping Beauty, Belle and Snow White.  Later, a cast member quietly asked us to come back downstairs because Cinderella had something special for the girls.  She was waiting for Brooke and Brielle with two little purses in her hand that matched the girls’ princess dresses.  Brooke and Brielle could not have been any more excited to spend more time with Cinderella!  As they opened the purses, they found their own little white gloves inside.  It was the sweetest gesture and they were so eager to put them on ASAP!  Cinderella even helped the girls put them on and was so sweet to take many more pictures after that.  That was another moment that made my heart swell with happiness.  We loved every second of this day and it only got better.  We had a special meeting with another favorite Princess… Rapunzel!! …and we were told that someone made reservations for us to have VIP seating for the night parade.  WOW! 

The parade was past Brooke’s bedtime so she stayed back in the hotel room with Grandma.  When we got seated and the floats started parading past, it seemed magical just because it was at night and everything was lit up.  We were so close to the action and some of the characters even came up to give Brielle a kiss on the hand.  She loved all of the attention and waved back at every single float that went by.  :)  One of the cast members came up to her before the parade started and gave her a little packet of pixie dust and said it was from Tinkerbell!  The cast member told Brielle to watch for Tinkerbell in the parade because she was going to blow her a kiss!  Sure enough as Tinkerbell went past she waved and blew kisses.  We all felt so special!  After the parade was over we got to keep our seats and stay for the fireworks show.  It was a perfect end to a more than perfect day.

After the parade, we decided to ride some of the rides at Magic Kingdom since the lines were so short!  Brielle loved all of them and she said her favorites were “The Mad Tea Party” tea cups, “Prince Charming’s Regal Carousel”, “Dumbo The Flying Elephant” and “It’s a Small World.”  We stayed out super late and we watched the clock strike midnight!

Day 4

Chef Mickey’s for breakfast was our first meeting with Mickey and Minnie Mouse!!  The girls absolutely loved meeting all the characters here and gave out many hugs and kisses!  The food was great too, the girls enjoyed Mickey Mouse shaped waffles, yum!

Dinner at the Garden Grill was another character meal starring Chip and Dale!  As the chipmunks were walking around we weren’t sure which was which until Brielle enlightened us that Chip has a brown nose like a chocolate chip and Dale’s nose is red.  It really surprised us that she knew that!!  This restaurant was really neat because it slowly rotated while you ate so you always had different scenery from the “Living with the Land” attraction and it finished up in the location where you started. 

Day 5

“‘Ohana’s Best Friends Breakfast with Lilo & Stitch”

We spent Day 5 in Animal Kingdom exploring in dinosaur land and playing the bongo drums in Africa.  We spent time looking for giraffes, lions, antelope, rhinos, warthogs, and zebras in the Kilimanjaro Safari ride. We were lucky enought to have front row seats at the “Finding Nemo” musical!  It was all so amazing!  On the monorail ride home,  Brielle spotted a cloud in the sky shaped like a dinosaur!

Day 6

We attended The Families of SMA (FSMA) Opening Night Meet & Greet with families and researchers.  They had a super fun carnival theme and the girls te too much popcorn, cotton candy, snowcones, etc.  :)  It was so much fun to meet other SMA families and researchers that are working so hard to find a cure!  Brielle raced against Dr. Kaspar (Gene Therapy Program at Nationwide Children’s Hospital at OSU) in her manual wheelchair and WON!  Yay Brielle!!

 Day 7

We spent the day at the conference and then had dinner at Norway’s Akershus Royal Banquet Hall in Epcot! 

Princesses Cinderella, Belle, Snow White, Sleeping Beauty and Ariel were there!  Brooke and Brielle even danced with the waitress.  :)

Rain, rain, go away!  It rained on the FSMA dessert/fireworks event at Epcot that night but it didn’t stop Brooke and Brielle!  They still visited Jasmine and Aladdin, got their faces painted and played with Children’s Hospital Boston friends in the alternate location inside!

 Day 8 - Fun in Epcot!

We had so much fun in Epcot that we just had to go back and spend another day after the conference sessions were over.  It rained again but we didn’t let that stop us.  The girls got to travel around the world and get stamps in their Epcot Passport and collected a button at the end!  They traded pins with cast members, got henna tattoos in Morocco, colored pictures in Italy and found Mulan in China.  We ended the day at “Mickey’s Backyard BBQ” and danced the night away with Chip & Dale, Minnie and Mickey, Goofy and Pluto!  At the BBQ, they asked all the kids to dance in the aisles with the characters.  We turned Brooke and Brielle around so they could dance in their chairs to watch all the fun and chipmunk Dale noticed them.  He came right over and danced with Brooke and Brielle for a really long time.  It was very sweet and they enjoyed dancing to the music.  It made our last day end on the best note.    

After all this magical fun, we were exhausted!!  It was an amazing trip and we wanted to make it as special as we could for our girls because we never know what the state of their health will be or the path that SMA will take our lives.  This has been a vacation and conference that we will all remember and cherish for the rest of our lives!  After we got home, Brielle said she wants to go back to Disney in 2 more days.  :)