Tell FDA to Save Our Children's Lives

Tell FDA to Save Our Children's Lives

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NPC Mamas started this petition to Food and Drug Administration

My name is Sara, and I am the mother of a child living with an ultra-rare and fatal disease called Niemann Pick Type C (NPC). Together with a group of patients and moms, we ask for your help in saving our kids’ lives. 

My six-year-old daughter, Marian, has NPC and is one of many children and adults relying on experimental medicines to keep living. But now, her medicine might get taken away. 

We are in a fight for our lives to get new therapies approved by the U.S. Food and Drug Administration, and we need your help. 

NPC is like Alzheimers, Parkinson’s and ALS rolled into one, except even more rare, causing extreme declines in how we move, talk, think, and even eat – pretty much everything. This disease occurs across all ages – and, horrifically, will frequently first show up in young children, even babies. 

There is no cure or approved therapy here in the United States, but there are several medicines that have been in development and used experimentally by patients for years and years. Patients AND our expert doctors see the tremendous benefits of these medicines in helping us. This is a BIG deal. It's life-saving. 

Heartbreakingly, the FDA has become a barrier to helping these therapies cross the finish line – restricting access, jeopardizing development, not listening to our expert clinicians, discounting patient benefits, and not understanding our ultra rare, variable, and fatal disease. 

The clock is ticking, and our kids will suffer with every day that goes by without action from the FDA.

The FDA has tried to keep new patients from accessing these experimental medications by erecting hurdles to early access programs, putting a clinical program on partial hold and denying requests for approvals. And we have had enough. This is putting TWO medicines patients need to live at risk of being taken away.

If our kids lose these medicines, they will die.

We need the following changes to occur immediately: 

  1. The FDA must lift all barriers creating life or death uncertainties for patients currently receiving investigational therapies for NPC. 
  2. The FDA must use regulatory flexibility and evaluate medications to treat NPC with the knowledge that NPC is ultra-rare, degenerative and fatal. 
  3. The FDA must immediately work with the scientific and patient community and design trials that are feasible, meaningful and do not put patients in harm’s way.

Sign and share this petition to tell the FDA that it must address the needs of those with NPC by approving medicines and increasing access to existing medicines.  This is urgent. Ours and our children's lives depend on this. Please help save our lives. Thank you!!

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