Community support

Patient organisations

Please find below a list of organisations that support the communities we seek to treat.

CLN2 disease

Batten Disease Support and Research Association (BDSRA) Australia

BDSRA Australia is the Australian Chapter of the Batten Disease Support and Research Association (BDSRA Australia). BDSRA Australia was founded in 1995 by parents of children affected by Batten disease. BDSRA Australia is dedicated to improving the well-being of patients and families affected by Batten disease in Australia.

Mucopolysaccharidosis (MPS)

Mucopolysaccharide & Related Diseases Society Aust. Ltd

The Australian MPS Society is a non profit organisation and was formed by a group of parents and friends in 1983, for those impacted by a range of rare genetic disorders known collectively as the mucopolysaccharide (or MPS) diseases. The MPS Society supports individuals affected with mucopolysaccharidosis (MPS), mucolipidosis (ML) and related disorders; parents of affected children; and other relatives and friends.  They provide connection, education and advocacy by holding conferences, family days and through social media.

Phenylketonuria (PKU)

Metabolic Dietary Disorders Association

The Metabolic Dietary Disorders Association (MDDA), a national self-help group, was founded in 1996 by parents to support other families and individuals affected by Inborn Errors of Metabolism (IEMs) which are rare genetic disorders (most diagnosed at birth via newborn screening such as PKU). The MDDA supports and represents families and individuals affected by a genetic Inborn Error of Metabolism whereby treatment involves a medically controlled diet.

The PKU Association of NSW

The PKU Association of NSW promotes awareness of PKU by providing information and support to individuals affected by PKU and their families;
facilitating interaction between individuals with PKU, families and health care providers; supporting research into the treatment of PKU, including fundraising for the purpose of research into the treatment of PKU; and lobbying Government bodies where appropriate.

Haemophilia A

Haemophilia Foundation Australia

Haemophilia Foundation Australia (HFA) is the national peak body that represents people with haemophilia, von Willebrand disease and other bleeding disorders, and their families. HFA aims to inspire excellence in treatment, care and support through representation, education and the promotion of research. HFA works with a network of State and Territory Foundations to ensure everyone with bleeding disorders in Australia has access to the world’s best practice treatment and care.

Rare Disease

Rare Voices Australia

Rare Voices Australia (RVA), the national peak body for Australians living with a rare disease, was established in 2012. RVA provides collaborative leadership for the development and implementation of rare disease policy in Australia. RVA is dedicated to working with all key stakeholders to drive the best outcomes for Australians living with a rare disease. RVA led the collaborative development of the National Strategic Action Plan for Rare Diseases which was launched by the Federal Minister for Health, the Hon Greg Hunt MP, in 2020.

Rare Disorders NZ

RDNZ is the respected voice of rare disorders in New Zealand and is the only national organisation supporting all New Zealanders who live with a rare condition, and the people who care for them. RDNZ offers a platform and connector hub with direct support to link people to information and rare disorder groups for details on specific rare diseases and to navigate the health system. RDNZ provides a strong unified voice to advocate for public health policy and a future healthcare system that works for those with rare disorders and works with the Government, clinicians, researchers and industry to promote diagnosis, treatment, services and research. RDNZ continues to advocate for a national framework for rare disorders in New Zealand.

Note: These listings are provided by BioMarin as additional information for patients, their families and their healthcare providers. The web pages and their content are maintained by the organisations listed above. With the exception of its own websites, BioMarin does not endorse any particular organisation or the content contained on their website.

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