About the Disease
Ceroid lipofuscinosis 2 (CLN2) is one of a group of rare genetic disorders called neuronal ceroid lipofuscinoses (NCLs), commonly known as Batten disease. CLN2, specifically, is a type of lysosomal storage disorder, in which individuals lack a specific enzyme that breaks down macromolecules such as lipids (fats) and proteins in intracellular compartments called lysosomes, which causes undegraded material to accumulate in neurons and other cells and leads to impaired cell function and neurodegeneration. Children with CLN2 disease rarely survive beyond their teenage years.
About our Patient Care
Our dedicated Care Team will work with the physician's office and insurance on a patient's behalf so that our patients have more time to focus on their treatment and quality of life. We are here to listen, provide
counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.
As part of treatment, we provide:
Dedicated Care Team
We provide prescribers and patients with a dedicated care team for each of our disease state programs. This includes a primary pharmacy contact, clinical education, side-effects management, scheduled refill calls, and a direct phone, fax and email address.
In-Home Patient- and Family-Centric Care
We administer a nationwide nursing network, allowing patients to pursue in-home treatment or receive training on self-administrated drugs. Our nurses are ideally positioned to establish a partnership with patients and their families in order to provide clinical education and coaching that strengthen the patient's capacity to achieve therapy goals.
Shipping and Coordination of Care
We provide free overnight delivery of medications and necessary medical supplies and manage the coordination of care between prescribers, sites of care, nurses and patients to ensure dispenses and drug delivery align with the date and location of service.
We successfully identify and assist enrolling patients into any and all manufacturer co-pay and foundation support programs in order to minimize a potential financial burden to patients given the high-cost of specialty medications.
24/7/365 Access to a Pharmacist
We provide prescribers and patients access to a pharmacist 24/7/365 to ensure patients are always cared for.
Brineura (cerliponase alfa)
Brand Name | Brineura
Manufacturer | Biomarin
Route of Administration | Intraventricular infusion
Approved Indication | Slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)
Self-Administration | No
Product Website | www.brineura.com