日前，美国罕见疾病协会正在全球范围内建立LAM病人的数据库，如果您是LAM病人，或者您身边有LAM病人，建议去协会网站上登记，以便能得到LAM研究及治疗的第一手信息，内容包括：您的发病日期、生日、邮寄地址、电话传真等个人信息，这些个人信息是绝对保密的，所以请大家放心。

网址是：https://rarediseasesnetwork.epi.usf.edu/rdnwebapp/registry/descriptionandpurpose.aspx?ownerid=7&diseasetype=36

附原文：

Hello Everyone,

I hope this finds you doing very well! I will soon get a letter to you regarding a few items I would like to discuss with you. For now, I want to let you know that the group conducting the MILES Trial, the Rare Lung Diseases Consortium (RLDC), has set up a website where you can register to receive information regarding the Trial. For those of you who may not have heard, the name of the trail has been changed from the SMILES Trial to the MILES Trial. MILES stands for Multicenter International LAM Efficacy of Sirolimus, sometimes called rapamycin. The website address for the RLDC is:  www.rarediseasesnetwork.org

Remember that this is simply signing the LAM patient registry to be contacted for information – not to enroll in the trial. By signing this registry you are committing to nothing more than being contacted as information becomes available. The registry is private. The only ones with access to the names are the members of the data control center in Tampa. They send out notices when the trial opens, when individual sites open, etc. To sign the registry to be contacted when information becomes available, please do the following:

1) Go to www.rarediseasesnetwork.org

2) Look for the heading that says “Rare Lung Diseases Consortium”

3) Under that you will see “Lymphangioleiomyomatosis (LAM).”

4) Click on that and it will take you to the page with information about LAM.

5) Scroll to the very bottom of the page and you will see Join the Contact Registry for Lymphangioleiomyomatosis

6) Click on that link and that will take you to another page.

7) Click on Join Contact Registry

8) That will take you to an agreement to read. Click on “I Agree” and that will take you to the form to register to receive information about the trial.

9) Complete the form and then click on “Register.”

You will be contacted about the trial as information becomes available.

I am sending this e-mail to over 600 people, so my concern is that I will be bombarded with questions that I won’t have time to respond to and/or don’t know the answers to! Please be patient – we are almost there! I know that you must have an FEV1 score of 70% or below to qualify for this particular trial, but I do not know where all of the trial sites will be. We are still awaiting approval from the National Institutes of Health (NIH). The Trial will begin at the University of Cincinnati and at the NIH first. Joel Moss will conduct the Trial at the NIH.

We are extremely anxious to get the Trial off the ground, so be assured that you will be informed immediately as information becomes available. You will receive a letter from the RLDC and also a letter sent first class from The Foundation. It will go to ALL women with LAM in our database. If you know of someone who has LAM who is not registered with the Foundation, please encourage them to register with us.

It’s been a long and arduous task. Dr. Frank McCormack, principal investigator for the Trial, has spent an enormous amount of time over the last 1½ years. Not only has he worked on the many details of the Trial, but he has written grants and acquired significant funding – the last success being a $200,000 commitment through the TSA Rothberg Courage Award. He has also written a grant to another source and we should hear about that soon. We are hoping to add more travel funds that will help to get patients to each of the sites. If we continue to be successful in acquiring funds from other sources, we can reserve Foundation funding for other research.

We remain excited about the Trial. We don’t know if this drug will work, but a controlled study such as this is the only way to find out! Thank you for your interest and than you for your patience!

Sue Byrnes, Director

The LAM Foundation

10105 Beacon Hills Drive, Cincinnati, OH 45241

Tel: 513-777-6889  Fax: 513-777-4109

E-mail: sbyrnes@thelamfoundation.org

Website: www.thelamfoundation.org