• Horizon Pharma Patients

Rare Diseases

Horizon Pharma is committed to getting patients and families the help they need to obtain ACTIMMUNE® (Interferon gamma-1b), RAVICTI® (glycerol phenylbutyrate) Oral Liquid and BUPHENYL® (sodium phenylbutyrate) tablets and powder.

The COMPASSSM Program (ACTIMMUNE)
The COMPASS (Comprehensive Personalized Patient Prescription Advocacy & Support Services) Program offers patients, their families, and healthcare providers one-stop convenient access to support services and to Program Coordinators who are available to answer questions about the Co-Pay Assistance Program, the Patient Assistance Program, and other patient support resources.

You or your doctor can enroll you in COMPASS. Once enrolled, you can access the following services at no cost.

You can find more information and support groups for people affected by CGD and SMO through the organizations listed below.

Horizon_Transcend_Rare_Logo_RGB
TranscendRareTM emphasizes our commitment and willingness to go the extra mile for all the patients we serve, with services and support that assist patients living with rare diseases and their caregivers.

For the general nephropathic cystinosis community, TranscendRare offers important information about nephropathic cystinosis as well as valuable tools to help you better manage the challenges along the way.

For people prescribed PROCYSBI, TranscendRare offers access and reimbursement support as well as ongoing nurse and e-mail communications to help you or a loved one stay on track with treatment.

For more information about PROCYSBI, please visit www.procysbi.com or call 855-888-4004.

UCD Support Services

Horizon Pharma is committed to supporting the urea cycle disorder community and will continue to make RAVICTI Oral Liquid and BUPHENYL tablets and powder available. We are also making the following support services available to all patients on Hyperion medications:

  • Access to financial support for eligible patients with high out-of-pocket costs for their UCD medication or for travel expenses to their UCD clinic
  • UCD medication at no cost for eligible patients with financial need
  • Support for patients searching for medical insurance

To access some or all of these offerings, or if you have any questions about your BUPHENYL or RAVICTI medication, please contact UCD Support Services at 1-855-UCD-SUPT (1-855-823-7878) or visit us online at UCDSupport.com.

We look forward to supporting the UCD community through these expanded offerings.

CGD Resources

Chronic Granulomatous Disease Association
A nonprofit corporation dedicated to helping CGD patients and their caregivers, and advancing research toward a cure.

Immune Deficiency Foundation (IDF)
The Immune Deficiency Foundation (IDF) is the national patient organization dedicated to improving the diagnosis, treatment, and quality of life of persons with primary immunodeficiency diseases, such as CGD, through advocacy, education, and research. IDF provides accurate and timely information and resources to patients and healthcare professionals.

CGD Society
Offers support and information to patients and families affected by Chronic Granulomatous Disease.

Global Genes | RARE Organization
Global Genes | RARE Project is a leading patient advocacy organization working to connect and empower rare disease patients and caregivers to become successful advocates by providing tools, resources and creating shared learnings to educate and inform. The Mission of Global Genes | RARE Project is to build and unify a global rare and genetic disease community while positively impacting patients in their lifetime.

SMO Resources

Ryan Wersten MIOP Foundation
The Ryan Wersten MIOP Foundation was created in memory of Ryan Wersten who lived for only six months after being diagnosed with Malignant Infantile Osteopetrosis (MIOP), also known as Severe Infantile Osteopetrosis. The Ryan Wersten MIOP Foundation is dedicated to supporting current research and to supporting families who have a child with MIOP.

Mason Shaffer Foundation
The Mason Shaffer Foundation is a nonprofit organization that has been dedicated to creating a support network for families of children with osteopetrosis.

The OsteoPETrosis Society (OPETS)
The OPETS is an organization that provides education and support to people diagnosed with osteopetrosis and medical professionals dealing with the disease. The Society also conducts informative sessions for patients through a Medical Advisory Council that provides expertise in the field of osteopetrosis.

Global Genes | RARE Project
Global Genes | RARE Project is a leading patient advocacy organization working to connect and empower rare disease patients and caregivers to become successful advocates by providing tools, resources and creating shared learnings to educate and inform. The Mission of Global Genes | RARE Project is to build and unify a global rare and genetic disease community while positively impacting patients in their lifetime.

Additional Rare Disease Resources

Global Genes – Allies in Rare Disease
Global Genes is a leading rare disease patient advocacy organization whose mission is simple: to eliminate the challenges of rare disease. They do this through developing educational resources, providing critical connections, and equipping advocates to become successful activists.

National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is a unique federation of voluntary health organizations 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.

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