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News and Events


 
 
April 20th, 2016

On April 20th, 2016, ALD Connect chair, Dr.Florian Eichler, presented bluebird bio's gene therapy study results. bluebird bio press release: bluebird bio Reports Interim Clinical Data from Starbeam Study of Lenti-D at AAN 2016 Annual Meeting

Xconomy also released an article on the study on April 20th: Bluebird's Gene Therapy Shows Promise in Rare Brain Disease

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ALD Connect 2016 Annual Meeting
 

November 2016

Boston, MA
 

*Please check back later for more details on the upcoming 2016 annual meeting

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X-ALD 101: Clinical Aspects and Screening Methods 

Webinar 

 
April 20, 2016 at 3:00-4:30PM ET
 
Dear Newborn Screening Community: 

In September 2015, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) recommended to the Secretary of Health and Human Services (HHS) to expand the Recommended Uniform Screening Panel (RUSP) to include X-linked Adrenoleukodystrophy (X-ALD). As most of you know, Secretary Burwell recently accepted this recommendation to expand the RUSP to include addition of X-ALD (and Mucopolysaccharidosis type I). 

Some newborn screening programs in the US are currently screening for X-ALD and others will follow in the coming years. There is a need for more information sharing to better understand the clinical aspects of X-ALD and available screening methods.

We are pleased to announce a webinar, "X-ALD 101: Clinical Aspects and Screening Methods" to be held on Wednesday April 20, 2016 from 3:00-4:30PM ET. This webinar is geared towards those who are or may be responsible for screening, diagnosing and treating X-ALD. This webinar is sponsored by APHL's Quality Assurance/Quality Control Subcommittee of the Newborn Screening and Genetics in Public Health Committee and the Newborn Screening Quality Assurance Program, Centers for Disease Control and Prevention.

The webinar will provide the audience with information regarding the clinical aspects of X-ALD inclusing an overview of the disease, available screening methods, follow-up and diagnostic evaluation, treatment options and available CDC Quality Assurance materials. Please find below the agenda for the webinar. 

CLICK HERE for webinar agenda and dial-in information.

If you have any questions, please contact Laura Russell at laura.russell@aphl.org, 240.485.2703


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ELA Meeting 2016

Families - Scientists 


April 02 & 03


The European Leukodystrophies Association (ELA) held its annual families and scientists meeting earlier this month, on April 2nd and 3rd in Paris, France. 
 
Below is a personal account of the ELA meeting by our Board member, Ben Lenail:
 
I just came back from the annual European Leukodystrophy Association (ELA) meeting in Paris. I attended the lunch and medical workshop on X-ALD on Saturday April 2. This was my fifth consecutive year attending.
 
The attendance at the ELA meeting is now at 500+. The meeting has moved to a new location, the Pullman Paris Bercy hotel, which has more space, better facilities for disabled folks, and better rates. The attendees are mostly French, with a good turnout from Belgium, Spain, Switzerland, Poland and many other countries.
 
I purposely sat with new folks during the lunch. Each family has a heart-breaking story. A mother had lost her daughter to metachromatic leukodystrophy at the age of 7 (after onset at the age of 4 and diagnosis at the age of 5). An elderly couple was taking care of their daughter, now 40 years old and in a vegetative state, who started showing severe neurological symptoms at the age of 32 from an undetermined leukodystrophy. The parents described how she had been a beautiful young woman, who was trilingual, excelled at the piano, and had a great career and many suitors.
 
Read full article...

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Adrenoleukodystrophy added to the

Recommended Uniform Screening Panel!


Feb. 17 2016

On February 17th, 2016, the Secretary of the Department of Health & Human Services, Sylvia Burwell, approved the addition of adrenoleukodystrophy (ALD) to the Recommended Uniform Screening Panel (RUSP), the federal list of genetic diseases recommended for state newborn screening programs. Read her letter here

The addition of ALD to the RUSP will expedite the process of adding ALD to state newborn screening programs. California and New Jersey can now join New York and Connecticut in screening newborns for ALD allowing for timely diagnosis and treatment.  Our work has only begun, however: there are still 46 states that need to begin ALD newborn screening as soon as possible.

The ALD community would like to thank Secretary Burwell and the incredible team of ALD advocates across the country for their support to help make this vision a reality. 

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Give up Your Data to Cure Disease 


Feb. 06 2016
 
"These digital databases offer an incredible opportunity to examine trends that will fundamentally change how doctors treat patients. They will help develop cures, discover new uses for drugs and better track the spread of scary new illnesses like the Zika virus...Medical research is making progress every day, but the next step depends less on scientists and doctors than it does on the public. Each of us has the potential to be part of tomorrow’s cures." Read article 

Click here to sign up for the ALD Connect Patient Portal. 

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Hurry up, Burwell, lives are at stake 

Feb. 04 2016
 
The Hill published an article on ALD being added to the Recommended Screening Panel.
Secretary Burwell has had 4 months to add ALD to the newborn screening list. What's the wait?  
California, New Jersey and Connecticut are ready to start. Babies lives are at stake! Please share and ask your friends and family to sign this petition! Read more 

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ALD Connect Patient Learning Academy

Saturday, February 20th, 2016


REGISTRATION NOW OPEN!


CLICK HERE for more infromation on

location, agenda & registration.



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ALD Connect Annual Meeting
 
October 01-03 2015

Boston, MA

 

The presentations given at our annual meeting are now ready to be viewed on our Annual Meeting page at http://aldconnect.org/2015-annual-meeting.php 
 

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CONGRATULATIONS!

At the hearing on 8.27.2015, the advisory panel voted 'YES'

to adding ALD to the Uniform Screening Panel!

 
 
HUGE THANK YOU TO ALL THOSE WHO WORKED SO HARD

TO MAKE THIS HAPPEN!

CHEERS!






Thank you again to all of our supporters!
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ALD Connect Patient Portal NOW AVAILABLE!!

Click Here to Sign Up



 
At ALD Connect, we have listened to you. We designed a Patient Portal where you can participate in a research study to create a global database of adrenoleukodystrophy (ALD) patients, their symptoms, and their priorities. We will be launching our Patient Portal soon, where you can register yourself and your family. On the Portal, you can answer survey questions about your health and help direct ALD research, so that together we can be one step closer to our ultimate goal; a cure. 

Who can participate in the portal? 

Have you been diagnosed with adrenoleukodystrophy (ALD) or adrenomyeloneuropathy (AMN)? Are you a parent of a child with ALD, or even a caregiver of someone with ALD? If so, we want you to join the Portal!

How will my participation in the portal help me?

 In the Portal, you will be able to track your abilities and health over time.

When we conduct research, the Portal will offer you the opportunity to learn about these studies first-hand and give you the ability to participate.

Your voice will be heard. With the Patient Portal, you will advance ALD research in the areas that matter most to you.

What will I find in the Portal?

As part of this study, you will find two simple tools:

What Matters to Me?
This questionnaire allows us to find out what health topic is of highest importance to you.

NeuroTracker:
This rating system allows you to rate your daily abilities on a scale and see how you are doing over time. 

Look out for additional questionnaires coming soon! 

How do I start?

Signing up is quick and easy! First, create an account with an e-mail and password. Next, review consent form and provide consent to participate in this research study. You can then decide which surveys you want to complete- each should only take a few minutes. We hope that you will continue to track your health over time in the Portal, but how much and how often you participate is up to you. 

How does my participation help ALD Connect?

ALD Connect wants to help advance research to benefit the patient community. By filling out our surveys, you help physicians and scientists truly understand where the real daily problems are and what to focus on. 

No Back Row Seats!

We want everyone to participate in the portal. Only you know how ALD has affected you and your loved ones. Please share this experience with us!

Thank you!

If you have any questions, comments, or concerns about the information presented to you, please contact ALD Connect at admin@aldconnect.org or at 617-643-4218.


 
ALD Connect Annual Meeting
 
October 01-03 2015
Boston, MA


 
ALD Connect will hold its 2015 annual meeting in Boston, MA.
Please check back later for further information on registration and meeting details. 

 
 

Past Events:
  • September 5-7, 2014, Palo Alto, CA: ALD Connect will have a presence at Stanford Medicine X with Ben Lenail and Keith Van Haren presenting ALD Connect.
  • September 13, 2014, Huntington Beach, CA: ALD Connect will have a presence at the Global Genes Project's 3rd annual tribute to Champions of Hope.
  • September 13, 2014 in Hoboken, NJ: Pig Roast benefiting the Aidan Jack Seeger Foundation.  The AJS Foundation was established to address the need for information and newborn screening with respect to Adrenoleukodystrophy. In addition, we strive to support and encourage those afflicted and their families as they struggle to endure, adjust and cope with the demands of the disease.
  • April 5, 2014, Paris, France: ALD Connect was present at the annual meeting of the European Leukodystrophy Association (ELA). Florian Eichler moderated a panel on ALD, and Florian Eichler and Ben Lenail presented ALD Connect during a keynote on "Patients as Drivers of Medical Research."
   
 
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