US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

Innovations in Care

We provide programs and services that empower the lives of people affected by neuromuscular disease.

Gene Therapy Community Support

MDA has invested over $125M in the development of gene therapy (GTx) for neuromuscular diseases over the past 20 years. With new gene therapy drug approvals in the pipeline, MDA is here to facilitate access and to provide support and education to the neuromuscular disease community.

Click here to connect with a MDA Staff!

How We Can Help


Contact Us

MDA GTx Support staff are available Monday through Friday 9 am to 5 pm CT. Answers to inquiries can be expected within 1-2 business days. MDA services are only available in the U.S. If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area.

By Phone: 1-833-ASK-MDA1 (1-833-275-6321)

By Email:

By Video: GTx Support Specialist Meeting
Meetings are video calls with an MDA GTx Support Specialist who can provide assistance locating resources, overcoming access barriers, navigating care within MDA’s Care Center Network and other healthcare providers, etc. to those eligible for approved gene therapies. MDA Specialists do not provide medical advice and/or counseling services.

Schedule Now

  • Schedule a Gene Therapy Support video call

    Step 1: Click here to schedule

    Step 2: Select MDA Staff

    Options are available to schedule with a specific MDA staff member, or select 'Anyone' for the broadest scheduling availability. MDA Gene Therapy Support video call appointments are available in English. To connect with an MDA Gene Therapy Support Specialist in another language please contact MDA's Resource Center at or 800-572-1717 to schedule a phone call utilizing a translation service.

    Step 3: Select Date & Time
    Gene Therapy Support appointments can be scheduled Monday – Friday 9AM – 5PM Central Time and are 30 minutes in length.

    Step 4: Your Details

    Please complete the form to fully book your appointment. Please include any details in the note section to better prepare your Specialist for the appointment.

    Step 5: Consent and Terms

    To participate in Gene Therapy Support you must be 18+ years of age and agree to MDA terms and conditions.

    Step 6: Review MDA Gene Therapy Support Video Call Confirmation Emails

    Once your appointment is booked, you will receive a confirmation email with the meeting link and details. You will be able to review, change, and/or cancel your booking via the confirmation email. Additionally, you will receive reminder emails prior to your meeting. Feel free to save the calendar invite to your personal calendar. Please note that appointments are scheduled in Central Time.

  • Preparing for Your Appointment

    Use these tips to help your 30-minute appointment run smoothly:

    • Check your email for MDA Gene Therapy Video Call instructions. Be sure to review any email communications from MDA. You will receive a confirmation email, appointment reminders, as well as updates about your upcoming appointment. Be sure to read these in advance.
    • Come prepared with your questions to make the most of your time with a Specialist. Please note that call recording is not permitted.
    • Reduce background noise. Try to find a quiet place free from distraction, if possible. For privacy purposes, headphones are recommended.
    • Closing other applications on your device can help facilitate a better video connection.
  • Joining Your Appointment

    Step 1: On the date and time of your appointment, please open the confirmation email you received and select the “Join your appointment” button, indicated by the red arrow. Please sign-in to your appointment at least 5 minutes prior to the scheduled start time to ensure the application is working properly.

    Note: You may need to download the Microsoft Teams App to participate in the meeting. If required, Microsoft will automatically prompt you and provide step-by-step instructions.

    Step 2: You will be asked to identify your name prior to the meeting.

    Step 3: You will be asked to confirm settings within your device to allow audio and visual features. In some situations, you may need to allow permission for your device to utilize your microphone and camera.

    Step 4: You will first be granted access into the meeting lobby, this space allows you to test audio and video features. The meeting will not begin until the MDA staff starts the meeting.

    Reminder: Your meeting time has been reserved specifically for you. Please arrive on time for your appointment. If you have not joined within 10 minutes of the meeting start time you will have to reschedule.

  • After Your Appointment

    Connecting with MDA doesn’t end when you log off! Continue to engage with MDA using these tips:

    • Schedule your next MDA Gene Therapy Video Call appointment. MDA is always here for you. We recommend connecting with a Specialist virtually twice a year.
    • Contact the National Resource Center. Call (800) 572-1717 Monday – Friday, 9AM – 5PM CT or email anytime for questions and support.
    • Your MDA Specialist will follow up with additional resources if needed after the appointment.
    • Give feedback and suggestions. Look for a survey to arrive in your inbox after your meeting. Feedback on your experience with your MDA Gene Therapy Video Call is appreciated.
  • Troubleshooting Technology

    Here are some common troubleshooting tips to use if you are having trouble logging in to your appointment:

    • Restart your computer or device.
    • Make sure your device is plugged in and charged.
    • Check your internet connection. Close additional applications to help improve connection. Sometimes connections are not strong enough to handle video streaming depending so turning off your camera and using voice may be best.
    • Update your device.
    • Try connecting with a different device.

    To connect directly with your scheduled Specialist by email, open up the confirmation email and/or calendar invite to collect the email of the Specialist you are assigned to send a notification email.

    • If you need to cancel or reschedule, feel free to do so at any time via the confirmation email or by calling MDA's National Resource Center at (800) 572-1717 or via email at
  • Friendly reminders...

    • All meetings are limited to 30 minutes.
    • By participating in a virtual appointment, you agree that you may not save, record, share or post this session or any photos from this session. MDA staff also agree that they will not save, record, share or post this session or any photos from this session. By participating in a virtual appointment, both parties agree to promote zero tolerance of hostile, violent or abusive behavior, consistent with MDA policies prohibiting intimidation and harassment.
    • If you are unable to keep this appointment and need to reschedule or cancel, please do so following the instructions in your confirmation email.
    • MDA Specialists are not medical professionals and will not provide medical advice and/or counseling services.

Request for Information: Connect with us here

Gene Therapy Clinician Support

MDA has invested over $125M in the development of gene therapy (GTx) for neuromuscular diseases over the past 20 years. With new gene therapy drug approvals in the pipeline, MDA is here to provide resources to the neuromuscular disease medical professional community.

Experts agree that although gene therapy holds great promise in addressing the underlying cause of many neuromuscular diseases, treatment of human patients requires clinicians to be prepared to address challenges and risks.

GTx Resources for Healthcare Professionals:

Contact Us

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