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  About ALD
About ALD Connect
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Our Organizational Structure & Leadership

  ALD Connect has a unique structure  
ALD Connect, Inc is an innovative marriage of ALD researchers and ALD families/advocates that whose goal is to rapidly improve the quality of life for ALD patients/families and eventually eradicate the disease entirely. To accomplish these tasks, ALD Connect has organized itself into two distinct networks, a clinical trial network and a patient network, designed to facilitate communication and innovation. These two arms of ALD Connect function in parallel, but can interact extensively through shared, focused communications (e.g. educational seminars to inform patients; surveys to inform researchers) and meetings.   

The "Commons" for Patients & Scientists @ ALD Connect
You are currently browsing the ALD Connect "Commons". This space is accessbile to both patients and scientists. It represents a crucial space for exchanging knowledge and ideas between ALD patients and scientists. Scientists from our clinical trial network will be able to share their insights and knowledge on how ALD symptoms occur, how to treat them, and what can be done to prevent them. Meanwhile, patients will be able to share their insights and priorities through our survey database which helps scientists design better treatments and better trials.

The "Clinical Trial Network" for Scientists @ ALD Connect

Our Clinical Trial Network (CTN) member sites, are medical institutions equipped to perform clinical trials in ALD/AMN. Each member site is required to:
   1. Have a lead investigator (researcher) who is responsible for the ALD Connect research activities
      at his/her institution.
   2. Have an out-patient clinic dedicated to caring for patients with ALD/AMN.
   3. Be certified in ALD/AMN outcome measures used to evaluate the results of clinical research.
View the CTN member sites.

The "Social Network" for Patients @ ALD Connect
ALD Connect will soon be opening an on-line social network exclusively for ALD/AMN patients, families, and advocates (i.e. a "PatientsLikeMe" or "MyHealthTeams" just for ALD). The site will be moderated by members of the ALD/AMN community and will allow patients and families the ability to organize, plan events, and share insights into the ALD experience with other patients and families. 

Most uniquely, the ALD Connect social network will also allow ALD patients and families an unprecedented level of input into ALD research. There are several ways this will happen. For example, ALD patients and families using the ALD Connect social network will be able to complete surveys (that are both voluntary and anonymous) that can aggregate and communicate their medical insights and priorities directly to scientists (e.g describe which symptoms matter most to women with AMN). This information will help scientists choose the best therapies and clinical trial options from among the many available possibilities. Currently, there is a long list of potential ALD therapies awaiting clinical trial opportunities. There are also many ways to design each clinical trial. Scientists need to know what symptoms or potential side effects matter most to patients. Scientists also need to know what kind of clinical trial design is of greatest interest to patients. Ultimately, scientists need to understand what matters most to patients in order to develop the best treatments.

We expect to open the social network to all members of the ALD patient community in mid to late 2014. To stay up to date on our progress toward this goal, join our email list or follow us on Twitter @ALDconnect.

Our Leadership

ALD Connect is governed by an Executive Committee made up of eleven members. Each member brings a depth of experience and a unique perspective to the governance of ALD Connect. Our research activities are advised by an experienced Scientific Advisory Board.

Board of Directors:
Josh Bonkowsky, MD PhD - ALD/AMN Clinician-Scientist @ University of Utah
Robert Brown, MD PhD - ALD/AMN Clinician-Scientist @ UMass Medical Center
Brian Chandler, Esq. - ALD/AMN Patient Advocate, based in Austin, TX
Florian Eichler, MD - ALD/AMN Clinician-Scientist @ Harvard/Mass General Hospital
Ali Fatemi, MD - ALD/AMN Clinician-Scientist @ Kennedy Krieger Institute
Stephan Kemp, PhD - ALD/AMN scientist @ Academic Medical Center, Amsterdam, the Netherlands
Ben Lenail - ALD Connect Business Strategy Officer, based in Palo Alto, CA
Kathleen O'Sullivan-Fortin, Esq - ALD/AMN Advocate from Boston, MA
Gerald Raymond, MD - ALD/AMN Clinician-Scientist @ University of Minnesota
Amber Salzman, PhD - ALD/AMN Advocate @ StopALD 
Alex Sherman - Clinical Database Specialist @ Harvard/Mass General Hospital
Keith Van Haren MD - ALD/AMN Clinician-Scientist @ Stanford University
Jennifer Farmer - Executive Director, FARA (Friedreich's Ataxia Research Alliance)

Data Coordinating Center

ALD Connect research and training activities are managed from our “Coordinating Center” located at Massachusetts General Hospital>>. The Coordinating Center is responsible for the management and conduct of ALD Connect-affiliated trials.

Learn more our Contracted Research Organization (CRO) capabilities.

What does it mean to be an “ALD Connect-affiliated” trial?

A trial is considered "ALD Connect-affiliated", when the sponsor of the trial has formally contracted with the ALD Connect Data Coordinating Center and Clinical Trial Network to help conduct the trial. A sponsor may contract with ALD Connect to manage an entire trial or just a portion of the work. To learn more, contact us at


Organizational Work Groups

Work Group Objectives Chair(s) & Members
Work Group #1: Infrastructure, Governance and Funding #1) Infrastructure and Resources for ALD
#2) Governance Structure and Operating Policies
#3) Consortium Funding and Project Opportunities

· Develop infrastructure and resources for ALD research and care.
· Create IRB/ICF templates.
· Establish ALD Connect as a legal non-for-profit entity.
· Establish by-laws that ensure participation of all stakeholders. Set up committees; data sharing & publication policies
· Highlight current opportunities that would align well with a focused initiative and directed funding
Board of Directors
Work Group #2: Outcome Measures for ALD #1) Outcome Measures
#2) Clinical Trials, Design, Challenges, Shortfalls
#3) Biomarkers

· Establish outcome measures to measure disease progression in research and clinical care.
· Explore novel and efficient self-reported data collection, including remote monitoring devices, mobile apps, and self-reported observations of daily living.
· Phase I trials models and preparedness; novel trial designs (adaptive, PK/PD)
· Status, gaps, how best to centralize, partnerships with industry, academia
Gerald Raymond

Work Group #3: 
Disease Mechanisms for ALD
#1) Disease Mechanisms for ALD
#2) Drug Development and Industry Perspectives

· Highlight potential mechanisms in ALD.
· Compare disease mechanisms from model systems with that of actual disease in humans.
· Prioritization of targets, industry standards and requirements for the ALD research field

Rachel Salzman & Kendrick Goss
Work Group #4:
Dissemination of ALD-related Disease Information
#1) Development of ALD-related Disease Information
#2) Patient Perspective and Patient-Centered Outcome Research
#3)  Healthcare-related costs of ALD

· Improve patient and physician access to ALD info. 
· Disseminate information on activity of consortium and research/trial activity (website, social media).
· Optimize guidelines for patient management.
· Study healthcare-related costs.
· Create Patient and Patient Caregivers Learning Institute and develop its curriculum.
· Establish pathways for patients to influence and guide research and clinical care strategies.

Keith Van Haren


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