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Fundraiser to get Muhammad Enzyme Replacement Therapy (ERT) for Pompe Disease
Current state:
- Muhammad is his parents first born, joining the world in April 2021. He has a story familiar to many in the Pompe Community - as his first months went by, symptoms that started out as whispers, soon began to yell. By October, genetic testing confirmed Muhammads Infantile Onset Pompe Disease (IOPD) diagnosis.
- With this diagnosis, time is critical! Traditional access to an ERT is not available to Muhammad in Pakistan, but of course his parents are going all in to help their baby boy!
Next steps:
- Muhammad needs to start treatment ASAP to have the best possible health outcome.
- Kids with IOPD who do not get treatment quickly, usually do not make it to their first birthday.
- Muhammads family is in touch with an industry partner currently producing an ERT for Pompe. They are working to enrol Mohammad in an Expanded Access Program (EAP) which will provide life saving treatment to little Muhammad, but this is expected to take some time to get in place - and with IOPD, his health is already declining so we don't have time to wait for the EAP program to kick in.
There is good news for bridging this time gap!
- There is a supply of an existing approved enzyme already in Pakistan that Muhammad will have access to. Based on his size this should cover ~6 months @ 20mg/kg biweekly
- However, the costs for the drug will have to be covered out of pocket. His parents are putting in everything they have, but those funds are limited so....
- We are rallying the community to support in raising funds to help cover the costs of starting ERT as soon as possible, and buy time for the EAP to go through.
Raising money to help kids diagnosed with infantile Pompe Disease in areas where they do not have traditional access to treatment. Starting treatment ASAP can save their lives and improve the health outcome for these kids. There are Expanded Access Programs (EAP's - offered through the pharmaceutical manufacturers of these treatments) to help these families receive treatment, but there is a time delay associated with applying, approving and administering the program. These funds will be used to cover out of pocket costs to receive ERT while the EAP is being set up (which can take several months).
If there are excess funds, they will roll over into a pool for future cases with similar need for bridging funds to assist families.
*Note, these funds are being raised through Rare Crossroads, a 501c3, to enable donations to be tax deductible (consult your local tax expert).
*Note, these funds are being raised through Rare Crossroads, a 501c3, to enable donations to be tax deductible (consult your local tax expert).
Organizer
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Rare Crossroads Inc
Beneficiary