The Save Our Sons Duchenne Foundation Nurses Program was developed to ensure “Best Practice” clinical care for all with Duchenne and Becker muscular dystrophy and facilitate bringing clinical trials to Australia and coordinate Australian-based clinical trials for persons with Duchenne and Becker muscular dystrophy.
The Nurses also provide coordinated care for Duchenne and Becker families, thereby improving standards of care across Australia. They coordinate with specialists to ensure that families attend all necessary appointments in an efficient manner and at the recommended intervals, in turn reducing the number of times families are required to visit the hospital. This helps ensure optimal care is provided and reduce the level of stress that families endure.
The Nurses have solid knowledge of each patient’s history, and also offer significant support to families navigating the complicated and difficult journey of caring for a child with Duchenne or Becker muscular dystrophy. They also provide specialist knowledge and experience with Duchenne and Becker muscular dystrophy to the multidisciplinary teams within hospitals and assist families to join the Australian Neuromuscular Disease Registry (ANMDR) to ensure critical and up-to-date data is available regarding the Duchenne and Becker population in Australia.
The Royal Children’s Hospital in Melbourne have over 570 neuromuscular (NM) patients, with the number of patients with dystrophinopathies (Duchenne and Becker) being just over 200 of this total.
The Save Our Sons Duchenne Foundation Board are very pleased to announce that they have allocated funding of $253,430 over three years towards a neuromuscular research nurse (Year 1 – $92,924.37; Year 2 – $84,476.70; Year 3 – $76,029.03).
The Royal Children’s Hospital Melbourne neuromuscular clinic operates weekly. Patients are seen in a multidisciplinary format, usually with 4 to 8 clinicians seeing each patient separately over the course of the relevant afternoon. At the end of the clinic, each patient is discussed in a multidisciplinary team meeting, during which management decisions are discussed at some length. The clinic generally runs for 5-6 hours.
The primary focus of this multidisciplinary clinic is best practice clinical care. A secondary focus is ensuring all children have access to new treatments for their condition as soon as these treatments become available in clinical trials or as proved therapies. Many clinic staff have both clinical and research roles, and many children are enrolled in clinical trials. During clinic visits clinicians always try to discuss available options for children who might be suitable for clinical trials and other research studies.
Roles part-funded through the Save Our Sons Duchenne Foundation in this neuromuscular clinic, in addition to the neuromuscular research nurse, include the neuromuscular clinical trials staff specialist neurologist and the clinic genetic counsellor.
Core responsibilities of the neuromuscular research nurse include:
- Communication with families regarding their child’s potential enrolment in a clinical trial
- Facilitation of consent procedures for clinical trials
- Preparation of submissions for ethics committee approvals
- Negotiations with clinical departments involved with clinical trial activities, including pharmacy, cardiology, medical imaging, respiratory medicine, laboratory services and the hospital paediatric pharmacology trials unit
- Scheduling of research visits including booking blood tests, scans, physician assessments and medication administration
- Completion of online data entry for clinical trials
- Meeting with representatives of clinical research organisations to ensure that all appropriate procedures are being undertaken.
The clinical trial activity of the neuromuscular team has increased to twelve active clinical trials (eight in DMD) with a total of more than fifty participants enrolled in neuromuscular clinical trials (with more than 35 boys with DMD).
The Royal Children’s Hospital in Melbourne are incredibly grateful for the support provided by the Save Our Sons Duchenne Foundation towards the roles of the neuromuscular research nurse, the neuromuscular clinical trials staff specialist, and the clinic genetic counsellor. This partnership is an example of a model for cooperation and collaboration between philanthropic organisations, clinical research units and private bodies committed to facilitation of significant research into potential new treatments for DMD.
In the near future an announcement will also be made in relation to an investment in setting up a new Australia-wide neuromuscular website with an associated Clinical Trial Finder which will provide a site for families and healthcare workers to find information about neuromuscular diseases, determine which clinical trials are running in different jurisdictions and identify which trials children might qualify for.