FYI - I just received this email from the folks gearing up for the AVi safety trial in Ohio. Lindsay Arnott is no longer with the institution, but they are up to date with their files.

Hello,

You contacted Lindsay Arnott who used to work in our institution, back in April and she had a chart open for your child. We have his name, date of birth and mutation and he is included in our list of potential candidates.
However, we are not ready to start the trial yet. The sponsor, AVI, will give his “go”, hopefully in a few months, and we will start enrolling patients. We will contact at this time to proceed to enrollment and screening. We would be very happy to have Liam in the trial and we will make sure to contact you back.
In the meantime, if you have questions about the trial, please feel free to email me.
Thank you very much.
Best regards,

Laurence

Laurence Viollet, PhD
Clinical Research Coordinator
Team Project Leader
Center for Gene Therapy
The Research Institute at Nationwide Children's Hospital
700 Children's Drive
Columbus, OH 43205
Phone: (614) 355-2695
Fax: (614) 355-5247
Laurence.Viollet@nationwidechildrens.org

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According to Dr. Leshner with CNMC - Prosensa will, in their next Dutch trial, be using a dose much higher than AVI. I know nothing about this trial or when it's going to be done. This is NOT in conjunction with any US related trial. I apologize for not clarifying that in any previous discussions. Once again, if I'm incorrect, I certainly don't mean to be and will, hopefully, be corrected in a friendly manner - not with guns blaring. At this point, all I have to listen to are Dr. Leshner and Eric Hoffman - and I must before a non-medical person on this site. Anyone has the option to read information presented by both AVI, Prosensa and any other drug company - as they obviously will present their best foot in order for folks to side with them. I have to take bits from all over - especially the kind people of this website. Hopefully, the doctors which treat DMD will come together and treat this disease as a whole, but I just don't get feeling that is happening right now.

I also feel that the Action Duchenne Conference at October's end of this year will be extremely positive as all the best minds in this field - from all over the world - will come together as they have not before to discuss exon 51 skipping. Who knows, maybe Pat will come away this weekend with something new. Maybe we can all then be on the same page, finally.

Ofelia Marin said:
I know.
Pat is attending this Treat-NMD meeting:

http://www.parentprojectmd.org/site/DocServer/092509_EMEA_briefing_...

I am looking forward to hear what they discussed especially about the "platform" drug concept.

irishgirl said:
The conference in London that I am referring to is the one hosted by Action Duchenne in late October.
Is anyone going to this london conference? I'm considering it
We are going - already booked! I think it will really be worth it!

jenn said:
Is anyone going to this london conference? I'm considering it
i will be there with my mother, my cell number is 802-579-7471, let me know if you want to hook up there, we will arrive thursday am, and depart sunday eve. i am excited to hopefully hear wonderful news!

irishgirl said:
We are going - already booked! I think it will really be worth it!

jenn said:
Is anyone going to this london conference? I'm considering it
Absolutely Jenn! Actually my hubby, Matthew Chambers will be going as I have to remain here to watch Liam. He will be arriving Friday morning in the early AM and departing on Sunday as well. I will give him your cell number and have him call when he arrives. His cell number is 703-623-3534 - to make sure you connect. He will be staying at the same hotel of the conference as well. It's great to have your Mom going with you. I really wish my mom were still alive, but just knowing her grandchild had DMD would be very hard on her.
Two minds at the conference will be much better than one!!!!! Great!!!!!!!

jenn said:
i will be there with my mother, my cell number is 802-579-7471, let me know if you want to hook up there, we will arrive thursday am, and depart sunday eve. i am excited to hopefully hear wonderful news!

irishgirl said:
We are going - already booked! I think it will really be worth it!

jenn said:
Is anyone going to this london conference? I'm considering it
i will look forward to meeting him, it is always good to be in the company of other parents who "get it". my mom has a very hard time emotionally, so im not sure how much she will be able to take in. she has never been to a conference, i always went alone to ppmd events, as i was a single mom... my husband craig ( my two oldest boys' step father) will be staying to care for our boys, along with my father and step father, we are a very close family. it will be a boys weekend for them, and will be the first time i have left all of them.it will be hard for me to go but i consider it very important to be in the know these days so, i will go to the ends of the earth if i have to! tell matthew to be sure to call when he arrives, and we will connect with him. take care, jenn

irishgirl said:
Absolutely Jenn! Actually my hubby, Matthew Chambers will be going as I have to remain here to watch Liam. He will be arriving Friday morning in the early AM and departing on Sunday as well. I will give him your cell number and have him call when he arrives. His cell number is 703-623-3534 - to make sure you connect. He will be staying at the same hotel of the conference as well. It's great to have your Mom going with you. I really wish my mom were still alive, but just knowing her grandchild had DMD would be very hard on her.
Two minds at the conference will be much better than one!!!!! Great!!!!!!!

jenn said:
i will be there with my mother, my cell number is 802-579-7471, let me know if you want to hook up there, we will arrive thursday am, and depart sunday eve. i am excited to hopefully hear wonderful news!

irishgirl said:
We are going - already booked! I think it will really be worth it!

jenn said:
Is anyone going to this london conference? I'm considering it
I get what you say, totally. Of course it will be hard to leave, but it will make you a better mother with more knowledge and that knowledge is power. I hope that you and Matt will come away from the conference feeling hopeful.
And can you tell me why people just don't get it?? I've explained DMD to Liam's current teacher twice and just last week, she had the audacity to say to me - and I quote, "Liam is AWLAYS two steps behind everyone in the class." Well, DUH!!!!!!! But, we are on the same page and I wish I were going as well to have the chance to meet you, but Matt retains information so much better than I do - I get too emotional - probably more like your mom!!!
It will be a wonderful weekend - except for the getting out and exploring London part - not too much time for that.
I'm glad you are out there on this site - where people do Get It!!! SO thanks, Jenn.

jenn said:
i will look forward to meeting him, it is always good to be in the company of other parents who "get it". my mom has a very hard time emotionally, so im not sure how much she will be able to take in. she has never been to a conference, i always went alone to ppmd events, as i was a single mom... my husband craig ( my two oldest boys' step father) will be staying to care for our boys, along with my father and step father, we are a very close family. it will be a boys weekend for them, and will be the first time i have left all of them.it will be hard for me to go but i consider it very important to be in the know these days so, i will go to the ends of the earth if i have to! tell matthew to be sure to call when he arrives, and we will connect with him. take care, jenn

irishgirl said:
Absolutely Jenn! Actually my hubby, Matthew Chambers will be going as I have to remain here to watch Liam. He will be arriving Friday morning in the early AM and departing on Sunday as well. I will give him your cell number and have him call when he arrives. His cell number is 703-623-3534 - to make sure you connect. He will be staying at the same hotel of the conference as well. It's great to have your Mom going with you. I really wish my mom were still alive, but just knowing her grandchild had DMD would be very hard on her.
Two minds at the conference will be much better than one!!!!! Great!!!!!!!

jenn said:
i will be there with my mother, my cell number is 802-579-7471, let me know if you want to hook up there, we will arrive thursday am, and depart sunday eve. i am excited to hopefully hear wonderful news!

irishgirl said:
We are going - already booked! I think it will really be worth it!

jenn said:
Is anyone going to this london conference? I'm considering it
AVI BioPharma Presents Updated Safety Data from Ongoing Systemic Trial of AVI-4658 at 7th Annual Action Duchenne International Conference

http://www.avibio.com/news_detail.php?newsId=0063

Data show PMO well tolerated in penultimate dose cohort (10mg/kg)


For Immediate Release
Bothell, Washington — October 26, 2009 — AVI BioPharma, Inc. (Nasdaq: AVII), a developer of RNA-based drugs, presented an update on preliminary safety data from its ongoing systemic Phase 1b/2 clinical trial of exon skipping AVI-4658 in patients with Duchenne muscular dystrophy (DMD) at the 7th Annual Action Duchenne Conference in London, UK.

The most recent data from the ongoing Phase 1b/2 trial at two MDEX sites in the UK demonstrate that AVI-4658 was well tolerated by DMD patients in a dose escalation study that is now up to the fifth cohort (10 mg/kg). Results from the 12 week dosing periods of the first four completed cohorts (0.5 mg/kg, 1.0 mg/kg, 2.0 mg/kg and 4.0 mg/kg) have been reviewed with data from the ongoing cohort 5 (10 mg/kg) and have demonstrated the drug to be well tolerated. There have been no safety issues identified, although one Serious Adverse Event was reported due to an anaesthetic-induced post-treatment biopsy procedure at 14 weeks, 2 weeks after last dose, causing nausea and vomiting.

The maximum cumulative dose administered to date is 2797 mg and the maximum single dose is 300 mg with no signs of intolerance, in either case. Blood tests, including measures of coagulation, have remained satisfactory, with the elevated levels of muscle enzyme, creatine kinase tending to fall in most boys during treatment. Lung function, vital signs and examinations have also remained stable. In addition, no significant side effects from the treatment have been reported for any of the 16 boys treated to date.

In each cohort, including the final cohort of 20 mg/kg, data for the clinical effects of the treatment will be collected for 26 weeks from first dose.

“We are pleased with the continued, encouraging safety profile and believe this bodes well for our ability to progress to the final cohort at 20 mg/kg,” said Steve Shrewsbury, M.D. Chief Medical Officer and Senior Vice President of Preclinical, Clinical and Regulatory Affairs. “We look forward to the continued advancement of this trial and to the potential that exon skipping may hold as a first disease modifying therapy for DMD patients.”

Also presenting at the Action Duchenne Annual Conference was AVI collaborator Steve Wilton, Ph.D. Professor at the Center for Neuromuscular and Neurological Disorders, University of Western Australia, Perth, Western Australia, Australia. Wilton presented an overview of exon skipping and clinical trials and also chaired a workshop on exon skipping.

“The Action Duchenne Annual Conference brought together leaders in the research and treatment of Duchenne muscular dystrophy from arounf the world. It is exciting to hear about AVI’s ground-breaking progress in its trial of AVI-4658 and the potential for exon skipping drugs to help the thousands of DMD patients who do not have an effective therapy for this disease,” said Nick Catlin, Chief Executive Officer of Action Duchenne.

The open label dose-finding clinical trial is evaluating the systemic delivery of AVI-4658 once per week for 12-weeks by intravenous infusion. Although the study is primarily a safety trial, it includes measures of drug efficacy and pharmacokinetics and is being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities by members of the MDEX Consortium led by Professor Francesco Muntoni and by Professor Kate Bushby at the Royal Victoria Infirmary, Newcastle-Upon-Tyne, UK, which is the coordinating center for the European Treat Neuromuscular Diseases (Treat-NMD) initiative. The clinical costs for the trial are provided, in part, by the UK Medical Research Council.

About Duchenne Muscular Dystrophy (DMD)

DMD is one of the most common fatal genetic disorders to affect children around the world. Approximately one in every 3,500 boys worldwide is afflicted with Duchenne Muscular Dystrophy with 20,000 new cases reported each year. It is a devastating and incurable muscle-wasting disease associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Symptoms usually appear in male children by age three. Progressive muscle weakness of the legs and pelvis eventually spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair by age 12. Eventually, this progresses to complete paralysis and increasing difficulty in breathing requiring ventilatory support. The condition is terminal and death usually occurs before the age of 30. The outpatient cost of care for a non-ambulatory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into development.

About the MDEX Consortium

The MDEX consortium led by Professor Francesco Muntoni, is a multidisciplinary enterprise to promote translational research into muscular dystrophies, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle University), and scientists from Imperial College London (Professor Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University of London (Professor George Dickson), Oxford University (Dr. Matthew Wood) and University of Western Australia (Professor Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participate in the Consortium. For more information, visit www.mdex.org.uk.

About AVI BioPharma
AVI BioPharma is focused on the discovery and development of RNA–based drugs utilizing proprietary derivatives of its antisense chemistry (morpholino-modified phosphorodiamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms of action. Unlike other RNA therapeutic approaches, AVI’s antisense technology has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA), allowing for both up- and down-regulation of targeted genes and proteins. AVI’s RNA–based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy as well as for the treatment of cardiovascular restenosis through our partner Global Therapeutics, a Cook Group Company. AVI’s antiviral programs have demonstrated promising outcomes in Ebola Zaire and Marburg Musoke virus infections and may prove applicable to other viral targets such as HCV or Dengue viruses. For more information, visit www.avibio.com.

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