Change.org - Approve our medication

Parents of children living with NPC are calling on the FDA to approve life-saving new therapies. “We are patients and parents of children and adults living with an ultra-rare and fatal disease called Niemann Pick Type C (NPC). We are in a fight for our lives to get new therapies approved by the US FDA, and we need your help.” Without a cure, these therapies are parents’ only hope of helping their children walk, talk, play, and swallow. Join these parents in demanding the FDA approve necessary medication for children with NPC. Tell FDA to Save Our Children’s Lives 12,194 have signed NPC Mamas’s petition. Let’s get to 15,000!

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Hi, my name is Sara and I am writing with a group of patients and moms of children living with an ultra-rare and fatal disease called Niemann Pick Type C (NPC).   My five year old daughter, Marian, has NPC and is one of many children and adults relying on an experimental medicine to keep living. But now, her medicine and another might get taken away. We are in a fight for our lives to get new therapies approved by the US FDA, and we need your help. The clock is ticking, and every day that goes by NPC is damaging us and our children. 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. FDA is keeping most 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:  The FDA must lift all barriers creating life or death uncertainties for patients currently receiving investigational therapies.  The FDA must use regulatory flexibility and evaluate medications to treat NPC with the knowledge that NPC is ultra-rare, degenerative and fatal.  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. Add your signature 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!! NOTE: To donate to our cause, please donate to www.gofundme.com/saveourmedicine. If you want to donate to the administrative costs for change.org the platform prompts you with that option after signing. This is optional and can be bypassed, those funds do not go to our NPC efforts.  You can follow our stories and fight on: Facebook Instagram Website #dontgiveuponnpc

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