Hi Julie, sorry for not being active here but I don't know why I cannot open the community page from my computer. So I have to use another computer. PLEASE come and see us, let's talk. You can also stay with us although I know you may be here already. Here is my phone number 513-765-9416. Here is my address: 6576 Carriage Hill Ln, Madeira OH (Exit 11/12 in I-71). Where are you staying?
My email is firstname.lastname@example.org. I would enjoy the article. That's great that your son is in cub scouts. We were not able to make the commitment. We go back to Cinci in July. We picked the summer months so we can skip out on the snow..just missed the blizzard last year by one day.
My son is doing well. We were in Cinci in July and go to Pittsburgh this Friday for a check up. I'm lucky he continues to do well in school except for a few behavior issues this past month. not sure why. How is Brandon? Anxious, Excited about exon skipping and all the promising news..just need it to get here FAST!!!!
Thank you so much. I work for Dr. Jude Samulski who is a leader in the research field of DMD. We are in the process of partnering with PPMD and the FACES program. We are excited about the research going on in this field.
If you want to see more on the work Dr. Samulski is doing go to genetherapy.unc.edu. Take care.
I wont be able to go tot he conf. This is the website. This is all I know so far. Also check out Charleys fund website. Charley as the same deletion and his parents have raised millions of $$.
Hey Julie! I am thinking my husband is going to go with me to the conference....it's taken a while to get much of an answer from him....sorry for being so slow. Have you found any other people who are going? We'd definitely be up for going to the sessions together and look forward to seeing you there! Good luck with your fundraiser at the Pizza Inn! That's sounds like a lot of fun! Hope all is well! Hugs to Brandon.
The "Nada Chair" is actually something created originally for sporting events, then office situations, and such (http://www.nadachair.com). It is basically a back pad and adjustable straps that come to knees on adults. These have been recommended for "long leg stretching" efforts, enhanced with simultaneous usage of AFOs. I am in the process of determining if insurance will cover these. They should, but may require written prescription from Doctor Wong or her team.
Things have been hectic with this huge initial push and I am just now getting around to responding to messages from other concerned DMD parents.
It sounds like you are in pretty much the same place as we are currently. Eric and I just got back from our visit to Cincinnati Children's. Most of what was received there was fairly well expected from research done to that point, but I think the experienced, total experienced care was worth the trip! We are getting the AFOs and Nada Chair for stretching, as well as the TB test necessary to begin Deflazacort treatment. We should be good to start that when the Deflazacort arrives towards the end of next week.
It helped that he could watch a movie. The first one was just a little stressful but the second one was easier. Would love to try and meet.
Dale just loves Legos. He can play on the floor for hours with his legos. He has such a great imagination.
I live in Gainesville Florida with my husband and two sons. My oldest is Lee, he is 9 and does not have Duchenne. Dale will be 7 on April 11. He was diagnosed last year right before his birthday. Our first visit with Dr Wong was last July. Dale started Deflazacort in August and we just went for a check up at the beginning of March. He is doing great.
Thank you for responding to my comment. I have a few questions for you. Does Dr. Wong have different treatments other than that of neurology local? Also does insurance cover this visit? Do you know any assistance we could apply for my ins. is Aetna but they have been difficult with just therapy...I am a nurse by trade and I tell you I deal with difficult news daily...but how different it is when its in your backyard:( Gainesville is about 3 hours away from tampa. I am gonna contact the hospital in ohio and see if I can get a appt. sometime August because I graduate RN school than and my time is free We currently treat with a local neurology. We go to the genetic dr Thurs. we are so stunned by this diagnosis we knew his as a genetic condition but do not have it in our family. I dont know i am just so confused, mad,so sad and sick sometimes to my stomach over this any advice you can give me would be great
Hi Julie, more than willing to share the room. I already booked my room but always interested on share costs and in fact, thinking the same way you are thinking now, I booked it asking for 2 queen beds instead of 1 king bed. So we are all set! :)
I live in Cincinnati, have 3 boys and my oldest, who is 4 has DMD. Would love to meet you when you come. You can also stay with us if you want. We live 15 min from the Hospital, very well located. I like our sons to have friends the same age and to have them make friendships that last for their life time. Also, I think it is great for your son to make the visit like a visit to "Diego" and not a visit to the Hospital...
Sorry I relayed the wrong message. I misunderstood Karens message to me. I guess I heard what I wanted to hear. I did alot of reaserch last night and found what I needed. I'm disappointed but still very hopeful.
The exon 51 skipping trials currently underway unfortunatly will not benefit our son's mutation. They need exon 50 skipped. I can assure you of this and have asked Dr. Flannigan and others at the conferences and this is the one they need skipped. Don't give up hope though as there is another trial that will hopefully start that targets our son's mutation. (hopefully in 2010) . This is in thanks to Charley's Fund. Here is the press release:
AVI BioPharma Receives Major Research and Development Grant From Charley’s Fund for Duchenne Muscular Dystrophy
$2.45 Million Will Fund Research and Product Development Program
PORTLAND, Ore., & SOUTH EGREMONT, Mass.--(BUSINESS WIRE)--AVI BioPharma, Inc. (Nasdaq:AVII), and Charley’s Fund, Inc., today announced that AVI has been awarded a $2.45 million research grant from Charley’s Fund, a nonprofit organization that funds drug development and discovery initiatives specific to Duchenne muscular dystrophy (DMD). This award, the largest grant ever made by Charley’s Fund and the largest received by AVI from a nonprofit foundation, will support a new product development program using proprietary exon skipping technologies developed by AVI and its partner, Ercole Biotech, Inc., to overcome the effects of certain genetic errors in the dystrophin gene. The award will allow AVI to accelerate its development of new therapeutics for DMD, a top priority for the company.
“The technologies developed by AVI and its collaborators have incredible promise in addressing the fundamental cause of DMD: the lack of functioning dystrophin protein,” said Benjamin Seckler, M.D., president of Charley’s Fund. “Exon skipping offers the greatest hope for thousands of boys whose futures are in question because of this aggressive disease. We are optimistic that this research collaboration will lead to a human clinical trial in the United States to treat this generation of children with DMD.”
DMD is a devastating and incurable muscle-wasting disease associated with specific in-born errors in the gene that expresses dystrophin. Dystrophin is a protein that plays an important structural role in muscle fibers. When dystrophin is missing or nonfunctional due to a mutation in the dystrophin gene, as it is in DMD, the result is membrane leakage and fiber damage, ultimately leading to degeneration and death of the muscle fiber. In two-thirds of DMD cases, the genetic mistake is hereditary, but one-third of cases arise spontaneously.
“The directors of Charley’s Fund take their mission to find a cure for DMD very seriously. We at AVI are also passionate about this goal and are extremely honored by their recognition of AVI’s progress with exon skipping technology,” said K. Michael Forrest, interim chief executive officer of AVI. “With excellent preclinical data in hand, we believe our ESPRIT exon skipping technology holds great promise for developing products to treat this devastating disease. We look forward to getting started on the research and product development program immediately.”
The focus of the funded program will be to select and develop a lead molecule, based on AVI’s proprietary NEUGENE® chemistry, that is designed to skip dystrophin exon 50 to restore the proper RNA reading frame and production of functional dystrophin in patients with a particular type of mutation. This therapeutic approach is similar to that of AVI-4658, in development to potentially treat DMD patients with mutations that could benefit from skipping exon 51 of the dystrophin gene.
Funding under the Charley’s Fund grant is anticipated over the next several quarters. The exon 50 candidate will be developed by AVI and its cross-licensing and collaboration partner, Ercole Biotech, under an agreement signed by the two companies in May 2007. The program brings together experts on exon skipping from around the world in a significant collaboration that may benefit the DMD population worldwide.
The technologies developed by Ercole and AVI allow manipulation of the RNA splicing process and the production by cells of clinically desirable variants of relevant proteins. AVI refers to its therapeutic approach as ESPRIT (Exon Skipping Pre-RNA Interference Technology). Ercole uses the term Splice Switching Oligonucleotide (SSO) in referring to its drug discovery platform to redirect mRNA splicing. AVI believes that its NEUGENE chemistry is particularly well-suited to modify RNA splicing.
Keep the faith! Hopefully we'll hear something soon!