Rare Disease Day is a global initiative coordinated and set up in 2008 by EURORDIS (European Organisation for Rare Diseases) and more than 65 national alliance patient organisation partners. The event has played a critical part in building international awareness as rare or uncommon diseases and disorders can affect anyone, at any age, around the world.

With over 300 million people around the world living with a rare disease, the day has established a vital focal point that enables work to progress on the local, national and international levels. Increasing education, knowledge and research is the way forward to improve treatments, preventative measures and better diagnosis techniques.

To read more about the event please visit the Rare Disease Day 2023 website.

Perth Blood Institute (PBI)

PBI is committed to supporting the rare blood disorder community, providing information about the latest studies and raising much-needed funds for research. Our clinical trials unit works with pharmaceutical companies towards treatments for rare diseases such as myelofibrosis.

Myelofibrosis is an uncommon form of bone marrow cancer that interrupts the body’s normal production of blood cells. The condition can cause widespread scarring in the bone marrow, leading to severe anaemia that can cause weakness and fatigue. Bone marrow scarring can also lead to a reduced number of platelets which are important blood-clotting cells. Click HERE to read more about myelofibrosis.

PBI currently has three clinical trials focusing on myelofibrosis. The aim of each trial is to assess the safety, tolerability and/or efficacy of different drugs and treatment options. To read more about these trials please visit our website, PBI Clinical Trials.

PBI Patient story

In 2021, Andrew was diagnosed with myelofibrosis. Andrew's haematologist from the Western Haematology and Oncology Clinic (WHOC) suggested he might benefit from a clinical trial for bone marrow cancer patients. After just over 12 months being on the trial, Andrew has shown positive blood test results.

Click HERE to read more about Andrew's story.

With continual financial support, we can equip our scientists to develop world-class research into rare blood disorders and enable PBI’s clinical trials unit to grow. If you would like to make a donation, please visit our website.

Donate - You can help to make blood disorders a thing of the past.