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Urgent: SMA Type 1 infant – Zolgensma access blocked due to cost
Hello everyone,
I am a father from Iran and caregiver of an infant with SMA Type 1.
Our doctor says funding must be secured before contacting Novartis for Zolgensma, which makes access impossible for our family.
Our case is registered in hospitals in Dubai and Qatar.
Has anyone here accessed:
• Early / Managed Access programs
• international treatment pathways
• advocacy or nonprofit support
Any guidance would mean everything to us.
Thank you.
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3 Replies
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Hello everyone,
Just following up in case anyone has experience with early or managed access programs for SMA Type 1, or international pathways for Zolgensma.
Any guidance or even a small direction would mean a lot to our family.
Thank you again for your time.
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Hi, Hosein. I don’t have any firsthand experience on this, and I know someone already mentioned contacting CureSMA. However, I do know that the SMA clinic I go to in Boston, Massachusetts handles out-of-country patients and provides treatment for those not living in the U.S. You could try contacting them as well. Here is their information.
https://www.childrenshospital.org/services/spinal-muscular-atrophy-program/contact-us
childrenshospital.org
Spinal Muscular Atrophy (SMA) Program | Contact Us
Contact the Boston Children's Hospital Spinal Muscular Atrophy (SMA) Program.
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Hi Alyssa,
Thank you so much for taking the time to respond and for sharing this information. I truly appreciate it.
I will definitely reach out to the Boston Children’s Hospital SMA Program to see if they may be able to guide us regarding international patients or possible pathways for treatment.
Thank you again for your kindness and support.
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