Help us help others
Hey friends,
can you do me a favor and help our sweet Jeremy
and his friends?
Save our kiddos in only 20 seconds.
💜
🧬 Children with Sanfilippo syndrome and other rare diseases don’t have time to wait.
The FDA has rejected a promising gene therapy,
despite admitting that the clinical data is strong.
Now, families may have to wait a year or more while their children continue to decline.
But momentum is shifting at the FDA and we have a small window to speak up.
📢 Sign this urgent letter to the FDA asking for a faster review and a meeting with families:
https://unc.az1.qualtrics.com/jfe/form/SV_0ivKzumYy787guq (also linked in profile)
Deadline: August 6
(I’ll post a direct link in my stories as well)
Sawyer was fortunate enough to be included in a
gene therapy trial when she was first diagnosed
(2.5yrs old) before brain damage started to occur.
Believe it or not,
It was such a blessing she received even a small dose of the drug.
It has stabilized her and helped slow down the progression of the disease.
Did it cure her?
No.
But the science has progressed over the past 7yrs and it’s promising.
Every child deserves life,
they deserve a chance.
Please help us convince the FDA of that.
🙏🏻
Your voice matters. Please sign and share.
#ApproveHopeNow #WalkTheTalkMakary #NoMoreDelays #RareDisease
Sanfilippo syndrome is a rare and devastating genetic disorder primarily affecting children, characterized by progressive neurological decline and severe cognitive impairment. This condition, falling under the umbrella of mucopolysaccharidosis type III, currently has no cure, making gene therapy a crucial breakthrough in treatment efforts. Gene therapy aims to correct the underlying genetic defect by introducing a healthy copy of the gene into the patient’s cells, potentially halting or slowing disease progression. In recent years, clinical trials have shown promising data, with some patients experiencing stabilization or slower symptom progression after treatment, as exemplified by cases like Sawyer’s, who benefited from early intervention. Despite solid clinical evidence, the FDA recently rejected a promising gene therapy treatment for Sanfilippo syndrome. This decision has caused great concern among families and advocates, as it means delays that could lead to irreversible damage in affected children. The urgency is underscored by the fact that children with Sanfilippo syndrome and other rare diseases have rapidly declining health and limited time to wait for new therapies. Advocacy groups are urging the FDA to expedite the review process and hold meetings with families to understand the critical needs more directly. Public participation is vital. By signing petitions and raising awareness using hashtags like #ApproveHopeNow, #NoMoreDelays, and #RareDisease, supporters can help shift momentum toward quicker approval and availability of gene therapies. This is not only a fight for those currently affected but also a step forward in making rare disease treatments more accessible. Stakeholders, including regulators, researchers, medical professionals, and families, must collaborate to accelerate innovation while ensuring safety and efficacy. Taking action now by signing and sharing the urgent letter to the FDA can make a meaningful difference. Your voice adds pressure on policymakers to prioritize these life-saving treatments, offering hope to children who cannot afford to wait.
