Rare diseases

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Unit 607

November 2023

Rare diseases

The purpose of this activity is to provide you with insightful information about the investigation and diagnosis of rare diseases, and the management and support of people with a rare disease and their families.

A disease or condition is considered rare if it affects fewer than one in 2000 people. Many rare conditions affect multiple organ systems and are chronic, serious and progressive. Around 80% are genetic in origin, with the remainder including autoimmune conditions, infections and cancers.

Although these conditions are individually rare, they are collectively common with approximately 8% of Australians having a rare disease. There are more than 7000 known rare diseases, with new ones being discovered regularly.

People living with a rare disease, and their families, often face common challenges, including diagnostic delay, poor health outcomes, limited care and support options, lack of research, psychosocial impacts and significant financial costs. So, while no health professional can be an expert in all rare diseases, there are common approaches to diagnosis and management that can assist in the care of people living with a rare disease and their families.


Learning outcomes

At the end of this activity, participants will be able to:

  • summarise indications and pathways for identifying and investigating undiagnosed rare disease
  • outline approaches for finding accurate and appropriate information on rare disease
  • discuss considerations for communicating about heritable rare conditions with sensitivity and respect
  • describe strategies for holistic, person-centred care for people living with rare disease and their families
  • explain the importance of connecting with centres of expertise and patient support organisations.

Case studies

Below is a list of the case studies found in this month's edition of check. To see how these case studies unfold and gain valuable insights into this months topic, log into gplearning to complete the course.

Margaret, aged 30 years, presents to you for a pre-conception health check. She reports she is generally fit and healthy but wants to know if there is anything else she should do or be aware of before she starts trying to conceive her first child with her partner Julian.

Cassandra has brought her daughter Layla, aged 6 years, to your clinic due to concerns with her nasogastric tube. She was recently discharged from the local hospital where she had one week’s admission under a gastroenterologist. This is your first time meeting Layla and Cassandra, as the family has recently moved to your area from regional Australia, having worked on several remote properties.

Lucas, a boy aged 16 years, has recently moved to the area and is seeking a regular general practitioner. He is quiet and fidgets with a toy during his appointment with you. His mother, Jackie, accompanies him and explains that he has an intellectual disability and has recently been diagnosed with a rare disease, Raynaud-Claes syndrome. Jackie would also like to discuss options for support for her and the rest of her family to help with the impacts of this recent diagnosis.

Raafe, aged three years, is Faraz and Leisha’s first child. They have just moved to your area from another city in Australia. Leisha brings Raafe to you to register as a new patient and for an influenza vaccination. You notice Raafe is not yet talking and appears rather unsteady on his feet. He has somewhat distinctive facial features, with low-set ears and a scar on his chest. Leisha asks if you could organise a referral for Raafe to a cardiologist at the local hospital.


CPD

This unit of check is approved for 8 hours of CPD activity (two hours per case). The 8 hours, when completed, including the online questions, comprise four hours’ Educational Activities and four hours’ Reviewing Performance.
 
Educational
Activities
4
hours
Measuring
Outcomes
0
hours
Reviewing
Performance
4
hours

Complete check online

To enroll in this check unit online: 

  1. Log into myCPD home page
  2. Select 'Browse' and search for 597727
  3. Select the course and register

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