FDA Save Our Medicine
FDA Save Our Medicine
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, these medicines might get taken away.
We are in a fight for our lives. We’re calling on the U.S. Food and Drug Administration (FDA) to use their regulatory flexibility granted to them, to find paths forward for approval of our treatments and continue access through compassionate use.
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 eat.
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. Patients AND our expert doctors see the tremendous benefits of these medicines in helping us. This is a BIG deal. It's life-saving.
But, access to both medicines is at risk of being taken away. The system surrounding medicines for our disease is broken and needs to be fixed immediately. We need the FDA to work with NIH, Industry and the scientific community to guarantee access to the medicines we need and operate on the same urgent timetable that we, as patients, face.
Dozens of notable physicians across the country who treat kids with NPC have signed a letter to Dr. Janet Woodcock, the acting commissioner of the FDA, urging her to make these treatments a priority. The clock is ticking and the FDA has the opportunity to be a hero to hundreds of families affected by NPC.
If our kids lose these medicines, they will die.
Sign and share this petition to tell the FDA to address the needs of those suffering from NPC today by using their regulatory flexibility to find paths for approving our medicines and increasing access to existing investigational medicines.
This is urgent. Our children's lives depend on this. Please help save their lives. Thank you!!
NOTE: To donate to our cause, please donate to www.gofundme.com/saveourmedicine