Don't Give Up on NPC
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THE FDA, MEDICAL COMMUNITY, AND POLICYMAKERS can help.

Only 10 percent of rare diseases have an FDA approved treatment.  FDA has the power to successfully address the challenges of medicine development for rare diseases, including NPC. 

White paper (NPC)

October 2021: We are the patients and parents of patients with an ultra rare and life-threatening disease who are being failed by the systems designed to spur development and expanded access to important treatments. Government, industry and the medical community must come together to protect these children who have a fatal and progressive neurodegenerative disease. 

Read White Paper

FDA Meeting

August 2021: In this program, expert clinicians, patients and family speakers shared their perspectives on the effort to find new treatments for NPC, including patient/caregiver preferences, risk tolerance, and opinions about use of the NPC clinical severity scale as a reliable and meaningful tool in clinical research. Our hope was that the FDA would hear our voices and recognize the critical need to be more flexible in reviewing drugs to treat NPC.

Letters to fda from NPC community

August 2021 Listening Session: Letters from the NPC community in advance of, and as follow up to a listening session with FDA, August 2021.

Read Letters

FDA MEETING

April 2021: In presentations by NNPDF, expert clinicians and patient advocates, the program highlighted many aspects of the severe and devastating impact of Niemann-Pick disease on patients, caregivers and communities. The session also outlines five areas of action that we hope the FDA and the broader research and treatment communities will support moving forward.

Meeting Summary

Letter from FDA to The Hill

September 2021

Read

FDA MEETING

March 2019: Video recording of the externally-led, Patient-Focused Drug Development Meeting with FDA in March 2019. 

View Recording

NPC VOICE OF THE PATIENT REPORT

March 2019: Summary report of the externally-led, Patient-Focused Drug Development Meeting with FDA in March 2019. 

Read Report

CLINICIAN LETTER TO FDA

August 2, 2021

Read

FDA Letter to npc Community

June 17, 2021

Read

NPC COMMUNITY LETTER TO FDA (ARIMOCLOMOL)

June 25, 2021

Read

NPC COMMUNITY LETTER TO FDA (ADRABETADEX)

June 25, 2021

Read

Letter to FDA, Mallinckrodt & Mandos from AZ Senators & Congresswoman

May 24, 2021

Read

NPC Community's Open Letter

May 2021

Read

ADRABETADEX TREATING PHYSICIANS LETTER TO FDA

April 22, 2021 

Read

media RESOURCES

NIH clinical trial for treatment of rare, fatal neurological disorder

NIH-led clinical trials suggests slowed progression of rare neurological disease

NIH-led clinical trials suggests slowed progression of rare neurological disease

View Study

NIH-led clinical trials suggests slowed progression of rare neurological disease

NIH-led clinical trials suggests slowed progression of rare neurological disease

NIH-led clinical trials suggests slowed progression of rare neurological disease

Read More

NPC Long-Term Study Results

NIH-led clinical trials suggests slowed progression of rare neurological disease

National Library of Medicine Results

See Results

National Library of Medicine Results

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

National Library of Medicine Results

View Phase 1-2 Trial

Niemann-Pick Type C Patient & Caregiver Voices

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

Read Report

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

Preferences for Treatment for Niemann-Pick Type C January 21, 2021

View Results

Niemann-Pick Type C: Timeline

Without their medication, our kids can't fight back.

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