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Information for Families

Information for Families

The National Niemann-Pick Disease Foundation supports individuals and families affected by Niemann-Pick Disease.  Our support covers a range of needs that occur at various stages of Niemann-Pick Disease.  We offer referrals to assist with diagnosis and treatment of NPD, guidance for care of individuals with NPD, assistance to families caring for loved ones with the disease, educational tools, and even information on insurance matters and legal issues.

This Web site is continually updated, but if you don’t find what you’re looking for, contact NNPDF.

The NNPDF is a 501(c)(3) nonprofit organization that funds research toward treatments and a cure for NPD and supports individuals and families affected by the disease.  We count on, and we’re grateful for, your support to do our work.

Niemann-Pick Type C and Social Security Disability

The Social Security Administration receives millions of claims for benefits each year.  This overwhelming number of applications results in backlogged disability claims, creating wait times of more than two years for some applicants.

Some conditions are more severe, making this wait very difficult for some individuals and families.  In response, in 2008, the Social Security Administration implemented Compassionate Allowances guidelines, listing 88 disabling conditions that qualify for expedited claim approval.  Instead of waiting months for a disability claim to be approved, applicants who have been diagnosed with one of these conditions may be able to qualify for benefits in a matter of weeks.

Niemann-Pick Disease Type C is one of the 88 conditions that qualifies for Social Security Disability processing under the Compassionate Allowances guidelines.

For more information, read this article from the Social Security Administration’s Disability Web pages.

[Apr 29, 2013 blg]

“Compassionate Use” 
Access to Investigational Drugs

The term “compassionate use” refers to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available. Drugs that are being scientifically tested but have not yet been approved by the United States Food and Drug Administration (FDA) are called investigational drugs. Being able to use one of these drugs when you are not in a clinical trial specifically designed to study that drug has many names, but is most commonly referred to as compassionate use.

Given the recent discussions about access to cyclodextrin, we thought you might like to read more about compassionate access to drugs.  Cate Walsh Vockley, Certified Genetic Counselor, prepared an article about compassionate use, including links to additional resources.   You can read the FYI article posted here.

[Oct 16, 2009 mem]

FYI:  Issues Regarding Curcumin Therapy

in Niemann-Pick Disease, Type C Patients

Use of various supplements in NPD patients is routinely discussed by families and is a challenging issue. There is often little data on the use of supplements in children and teens, or even in adults, for that matter. There is also limited data on reasonable dosing and potential side effects.

One such supplement, curcumin, has been discussed over the last few months on the NNPDF list serv.  There have also been concerns raised in the scientific community that higher doses of this compound are not as effective as lower doses, and in fact, may be toxic rather than being helpful to patients.

Although there is no current data on the use of curcumin in NPC patients, click here for a summary of the limited data on the use of curcumin.  This information is not intended to be a medical directive or endorsement of the use of any product, but rather is for use by families as they consider the issue of curcumin supplementation for their affected family member.

[Oct 14, 2009 mem]

New Resource Available from NNPDF

Dementia Booklet

Dementia is something usually associated with older people. However, children, teenagers and young adults can experience dementia as a result of a number of rare diseases and conditions. Niemann-Pick Disease Type C (NPC) is an example of one of these diseases, and one of its main symptoms is dementia.

The NNPDF now has available a booklet for families, teachers and caregivers, explaining how dementia may be exhibited in the Niemann-Pick Disease patient, and offering practical tips for dealing with the related challenges.  Click here to read the new U.S. version of the Dementia booklet, modified from the booklet originally published in the U.K.

Contact the NNPDF if you would like to receive a copy of the booklet by mail.

[Aug 27, 2009 mem]

Comprehensive NPC Booklet for Physicians

The NNPDF has available a comprehensive booklet about NPC, written with the physician and other health care providers in mind. The booklet, called “Understanding Niemann-Pick disease type C and its potential treatment,” was written by Jackie Imrie, Clinical Nurse Specialist, and Dr. Ed Wraith, both of the United Kingdom NPD program.    A generous grant from Actelion Corporation sponsored the publication..

The booklet includes a summary of our current understanding of the disease processes based on research, as well as a summary of the clinical symptoms of the disease, genetics, diagnosis and treatment strategies. Please note: this publication is designed for health care providers so may be a bit technical for some families. Our intent is that you share it with your child’s health care team.

If you would like a copy contact the NNPDF, being certain to include your mailing address.  A limited number of copies are available.