Patients and Families

Important Safety Information
Product Information

Living with Hunter Syndrome

MPS/ML Forum Dot Com

A message board for those caring for a special needs child, specifically those with MPS/ML Disorders.
www.mpsforum.com

Alliance of Genetic Support Groups

Contains newsletter, resources, and forum for anyone affected by a genetic disease. The alliance promotes advocacy, education, and empowerment for genetic support groups.
www.geneticalliance.org

Children Living with Inherited Metabolic Diseases (CLIMB)

Information and advice for those, particularly children, affected by an inherited metabolic disease.
www.climb.org.uk

Lysosomal Storage Disorders Support

An online forum about lysosomal storage disorders.
www.lsdsonline.com

Alliance Sanfilippo

Alliance Sanfilippo is a nonprofit organization of parents committed to accelerating the pace of biomedical research and developing clinical trials in Sanfilippo Syndrome / MPS III. Founded in 2005 by parents with affected children, the organization aims at becoming a leading provider of support for MPS III research and resources throughout Europe.
www.alliancesanfilippo.com

Other Resources

Medicaid Waiver Program

www.cms.hhs.gov/MedicaidStWaivProgDemoPGI/
Optional program that may be offered in a particular state that can provide Medicaid specifically for home and community-based services for disabled children; may cover therapy services, case management, adapted equipment, aide services, and respite care. Visit www.cms.hhs.gov/medicaid to determine state program regulations.

US Department of Education

Information on the Individuals with Disabilities Education Act (IDEA) and The Rehabilitation Act.
www.ed.gov/policy/speced/leg/edpicks.jhtml?src=ln

Other Resources

www.disabilityinfo.gov
Resources regarding employment, education, housing, transportation, income support, technology, and community support.

Disabled Children's Relief Fund

www.dcrf.com
Helps children without adequate health insurance, especially the physically challenged, provides disabled children with assistance to obtain assistive devices, rehabilitative services, surgery, and support for arts and humanities projects.

Angel's Hands Foundation

www.angelshands.com
Angel's Hands Foundation's mission is to improve the quality of life for individuals living with rare diseases. It supports families by assisting with lingering medical expenses, funding medical equipment not covered by insurance, funding family visits to educational events or to see medical specialists, sponsoring fundraising events, and hosting social outings for families.

National Dissemination Center for Children with Disabilities (NICHCY) - Home Page

http://www.nichcy.org/
NICHCY stands for the National Dissemination Center for Children with Disabilities. They serve the nation as a central source of information on:

  • Disabilities in infants, toddlers, children, and youth
  • IDEA, which is the law authorizing special education
  • No Child Left Behind (as it relates to children with disabilities)
  • Research-based information on effective educational practices

A Parent's Guide:

Accessing Programs for Infants, Toddlers, and Preschoolers With Disabilities

www.nichcy.org/pubs/parent/pa2.htm

www.nichcy.org/pubs/spanish/pa2sp.htm en Espanol

This guide from the National Information Center for Children and Youth with Disabilities (NICHCY) is intended to assist families in obtaining help for their young children with special needs (ages birth through 5 years). It answers commonly asked questions about early intervention services for infants and toddlers (birth to 2 years), and related services for children ages 3 through 5 years old. This guide identifies the early intervention policies and contacts in your state and area.

NICHCY

State Resources for disability-related services
www.nichcy.org/states.htm

These resource sheets will help you locate organizations and agencies within your state that address disability-related issues, including:

  • State agencies serving children and youth with disabilities
  • State chapters of disability organizations and parent groups
  • Parent training and information projects
  • And much more, including the official State Web site, Governors and U.S. Senators, and other useful associations and organizations.

Healthcare Funding Resources

Medicare

Medicare is the national health insurance program for:

  • People age 65 or older
  • Some people under age 65 with disabilities
  • People with End-Stage Renal Disease (ESRD), which is permanent kidney failure requiring dialysis or a kidney transplant

To learn more about Medicaid and other programs available in your state, click here: www.cms.hhs.gov/medicaid/consumer.asp

To find out more by phone, you can call: 1-800-MEDICARE (1-800-633-4227)

For general Medicare information, ordering Medicare booklets, and information about health plans, contact 1-800-MEDICARE 24 hours a day, 7 days a week for assistance. English and Spanish-speaking customer service representatives can answer questions about Medicare and provide up-to-date information regarding the health plans available in your area.

If you already have Medicare and would like to find out more, click here:
www.medicare.gov/

Medicaid

Medicaid is state-administered health insurance that helps many people who can't afford to pay for medical care for some or all of their medical bills.

Medicaid is available only to certain low-income people and families who fit into an eligibility group that is recognized by federal and state law. Medicaid does not pay money to you; instead, it sends payments directly to your healthcare providers. Depending on your state's rules, you may also be asked to pay a small part of the cost (co-payment) for some medical services.

To learn more about Medicaid and other programs available in your state,
click here: www.govbenefits.gov

At the link above, click on your state for a listing of available programs.
GovBenefits.gov is a partnership of federal agencies with a shared vision - to provide improved, personalized access to government assistance programs.

MPS Society Resources

Family Assistance Program

The MPS Society provides grant funding for parents of a child with MPS/ML or an adult with MPS/ML. Those who reside in the US and are members in good standing with the MPS Society are eligible.
www.mpssociety.org/sup-fap.html

Advocacy

Family Voices

An organization that speaks on behalf of children and youth with special healthcare needs
www.familyvoices.org

Partners in Making Your Case

A self-study course in advocacy
www.partnersinpolicymaking.com/makingyourcase

SNAP (Special Needs Advocate for Parents)

Providing information, education, advocacy, and referrals to help parents ensure lifetime care and quality of life for their children with special needs
www.snapinfo.org/home.html

Wrightslaw

Accurate up-to-date information about special education law and advocacy for children with disabilities
www.wrightslaw.com

Especially for Kids

Taryn's World

A special place for special kids
www.tarynsworld.org/taryn/index.htm

Especially for Parents

Exceptional Parent Magazine

Provides information, support, ideas, encouragement, and outreach for parents and families of children with disabilities, and the professionals who work with them
www.eparent.com

Family Village

A global community of disability-related resources
www.familyvillage.wisc.edu/index.htmlx

General Information

National Organization for Rare Disorders (NORD)

NORD is dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. Here you'll find a rare disease database, an online community, news, and event listings.
www.rarediseases.org

National Society of Genetic Counselors

Understand why genetic counseling is important and find a genetic counselor in your area.
www.nsgc.org

National Institutes of Health - National Human Genome Research Institute Glossary

Glossary of genetic terms for non-scientists
www.genome.gov/glossary.cfm

ClinicalTrials.gov - An Introduction to Clinical Trials

Answers to frequently asked questions about clinical trials for patients
www.clinicaltrials.gov

Indication
ELAPRASE® (idursulfase) is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). ELAPRASE has been shown to improve walking capacity in these patients.


Important Safety Information


Some patients in the clinical trials experienced life-threatening immediate allergic reactions to ELAPRASE (idursulfase) infusion. Patients who have experienced severe allergic reactions may experience another allergic reaction approximately 24 hours after the initial reaction and may require prolonged observation. Patients with compromised respiratory function or acute respiratory disease may have a higher risk of life-threatening reactions to ELAPRASE infusion and require additional monitoring.


Life-threatening allergic reactions have been seen in some patients during ELAPRASE infusions. Reactions included difficulty breathing, lack of oxygen, seizure or loss of consciousness, hives, and/or swelling of the throat or tongue. Patients received an antihistamine to reverse the allergic reaction or corticosteroid to decrease inflammation prior to or during later infusions. ELAPRASE was given more slowly or was stopped early if patients had serious reactions. With these measures, no patients stopped treatment permanently due to an infusion reaction.


Because of the potential for life-threatening complications, the ELAPRASE infusion may have to be delayed for patients who have respiratory illness and/or fever.


The most common side effects that required intervention had to do with infusions. These included headache, fever, rash, hives, itching and increased blood pressure. These side effects decreased over time with continued ELAPRASE treatment.


In clinical trials, patients given ELAPRASE once a week for 52 weeks had the following side effects at a rate greater than placebo and at least 10%. Approximately:


About half of the patients in clinical studies produced antibodies to treatment with ELAPRASE and these patients had an increase in infusion reactions. The presence of antibodies on the effectiveness of ELAPRASE is unknown.

Please see full Prescribing Information including Boxed Warning