{"id":196,"date":"2024-11-20T18:51:30","date_gmt":"2024-11-20T18:51:30","guid":{"rendered":"https:\/\/www.brineura.com\/en-us\/?page_id=196"},"modified":"2026-03-12T13:45:27","modified_gmt":"2026-03-12T13:45:27","slug":"getting-started","status":"publish","type":"page","link":"https:\/\/www.brineura.com\/en-us\/getting-started\/","title":{"rendered":"Getting Started"},"content":{"rendered":"
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Discover the path toward treatment with Brineura® (cerliponase alfa)<\/h1>\n\n\n

Every child’s journey with Brineura® (cerliponase alfa<\/span>)<\/span>, an enzyme<\/span> replacement therapy, will be different. Learn about how to get started.1<\/sup><\/p>\n\n\n <\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Your doctor will determine if Brineura is right for your child<\/h2>\n

DOCTOR’S APPOINTMENT<\/strong><\/p>\n

You’ll consult with your child’s doctor and, together, determine whether Brineura is right for your child.<\/p>\n

Your child’s doctor may consult with other experts more familiar with treating CLN2 disease<\/span><\/span>. You and your child may need to travel to one of these experts at a different hospital to receive treatment.<\/p>\n <\/div>\n \n

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Roadmap to Brineura\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Your healthcare team at the hospital will work on an individualized plan for your child, which your doctor will discuss with you. This roadmap is meant to give you an overview of the path toward treatment. Your family may experience a similar path, or your path may vary.<\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

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Download a copy of this roadmap for your family<\/a><\/p>\n <\/div>\n\t\t\t\t\t <\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Your doctor will create a treatment plan specific to your child’s needs<\/h2>\n

HOSPITAL TREATMENT PLAN<\/strong><\/p>\n

Brineura is a specific therapy, so creating a treatment plan specific to your child’s needs may take some time.<\/p>\n

Brineura requires a multidisciplinary team—that means many people from different departments in the hospital will be involved. The hospital will work with your insurance provider to establish reimbursement for Brineura therapy.2<\/sup><\/p>\n

As a caregiver, you’re the most important part of your child’s team. You are your child’s advocate and a key source of information, whether it’s medical records or insights into your child’s well-being.<\/strong><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Matty is a patient with CLN2 disease.<\/h2>\n

He’s been on treatment with Brineura since 2015.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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On infusion<\/span> days: “Matty loves his iPad and enjoys playing games, looking at books, and watching movies on it. The iPad makes life so much easier for him (and us) in the hospital.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t —Joe, Matty’s dad<\/span><\/cite>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/blockquote>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Before starting Brineura<\/h2>\n

Brineura is administered every other week through intraventricular infusion<\/span><\/span>.1<\/sup> This method allows Brineura to be delivered directly to a ventricle<\/span><\/span> in the brain, and then into the fluid surrounding the brain, known as the cerebrospinal fluid<\/span> (CSF)<\/span>. Brineura is delivered into the CSF<\/span> to help reach cells that are affected by CLN2 disease<\/span>. Knowledgeable members of your healthcare team will give your child’s Brineura infusions.<\/span>1<\/sup><\/p>\n

To receive intraventricular infusions<\/span>, your child will first need to have an intraventricular access device<\/span><\/span> surgically implanted.1<\/sup> This is an established procedure in pediatric neurosurgery.2<\/sup> The neurosurgeon will discuss the procedure with you and answer any additional questions you may have.3<\/sup><\/p>\n

Your healthcare team will let you know how to prepare your child for infusion<\/span>, and what to expect during this procedure. Your child will be monitored before, during, and after the infusion<\/span>, and may receive medications to reduce the risk of hypersensitivity<\/span><\/span> reactions.1<\/sup><\/p>\n

Low blood pressure and\/or slow heart rate, and undesirable hypersensitivity reactions, including fever, vomiting, irritability, and anaphylaxis, may occur during and following the Brineura infusion. Contact your healthcare team immediately if any occur.1<\/sup><\/p>\n

After your child has been prescribed Brineura, some of the steps may include:<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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MRI brain scans3,4<\/sup><\/h5>\n

MRI*<\/span> scans are used to help the surgeon locate where the intraventricular access device<\/span> should be inserted and to confirm placement after surgery.<\/p>\n

*MRI, magnetic resonance imaging.<\/small><\/p>\n <\/div>\n<\/div>\n\n

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The intraventricular access device will be surgically implanted<\/h5>\n

This established procedure is necessary for your child to receive Brineura.1,2<\/sup> It allows direct delivery of Brineura into a ventricle<\/span> in the brain.<\/p>\n <\/div>\n<\/div>\n\n

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Brineura infusions can begin1<\/sup><\/h5>\n

It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.1<\/sup> Work with your healthcare team to schedule treatments.<\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Brineura treatment will take approximately 2 to 4.5 hours, depending on the dose and volume administered, every other week.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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BioMarin RareConnections™ Support Services\n<\/h3>\n

Experienced financial navigation support<\/strong><\/p>\n

BioMarin RareConnections provides families with one-to-one financial navigation and logistics support to start and continue therapy.<\/p>\n

Learn more about our support services at BioMarin-RareConnections.com<\/a>.<\/p>\n

Learn more<\/a><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Enroll your child in BioMarin RareConnections™<\/h5>\n \n <\/div>\n
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RareConnections<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Brineura is the only treatment that directly addresses the underlying cause of CLN2 disease<\/h5>\n \n <\/div>\n
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About Brineura<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Connect with BioMarin to register for Brineura updates<\/h5>\n \n <\/div>\n
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Register now<\/a><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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References:\n<\/h4>\n\t\t\t\t\t\t
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  1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n
  2. Mole SE, Schulz A, Badoe E, et al. Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients. Orphanet J Rare Dis<\/em>. 2021;16(1):185.\n<\/span><\/li>\n
  3. Cohen-Pfeffer JL, Gururangan S, Lester T, et al. Intracerebroventricular delivery as a safe, long-term route of drug administration. Pediatr Neurol.<\/em> 2017;67:23-35.\n<\/span><\/li>\n
  4. Vuillemenot BR, Kennedy D, Cooper JD, et al. Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis. Mol Genet Metab<\/em>. 2015;114:281-293.\n<\/span><\/li>\n <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-196","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nGetting Started - BioMarin Brineura Patient EN-US<\/title>\n<meta name=\"description\" content=\"Every child\u2019s journey with Brineura\u00ae (cerliponase alfa), an enzyme replacement therapy, will be different. 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