Inherited retinal diseases (IRDs) are a broad range of diseases associated with abnormalities/degeneration of retinal cells. We aimed to identify the top 10 Australian research priorities for IRDs to ultimately facilitate more meaningful and potentially cost-effective research.

We conducted a James Lind Alliance priority setting partnership that involved two Australian-wide surveys and online workshops.

Australia-wide.

Individuals aged 16 years or older were eligible to participate if they had an IRD, were caregivers of an individual with an IRD or were health professionals providing care to this community.

In Survey 1, we gathered participants’ unanswered questions about IRDs. We grouped these into summary questions and undertook a literature review to verify if they were truly unanswered (ie, evidence uncertainties). In Survey 2, participants voted for the uncertainties that they considered a priority. Top-ranked uncertainties progressed for discussion and final prioritisation in two workshops.

In Survey 1, we collected 223 questions from 69 participants. We grouped these into 42 summary questions and confirmed 41 as evidence uncertainties. In Survey 2, 151 participants voted, with the 16 uncertainties progressing to final prioritisation. The top 10 priorities, set by the 24 workshop participants, represented (1) treatment/cure; (2) symptoms and disease progression; (3) psychosocial well-being and (4) health service delivery. The #1 priority was for treatment to prevent, slow down or stop vision loss, followed by the #2 priority to address the psychological impact of having an IRD.

The top 10 research priorities highlight the need for IRD research that takes a whole-person, systems approach. Collaborations to progress priorities will accelerate the translation of research into real-world benefits.

Guidelines for symptomatic knee osteoarthritis (OA) dictate the initiation of conservative treatment (physical therapy, analgesics and intra-articular injections with corticosteroids) as a first line defence. When conservative treatment fails, the golden standard is invasive joint replacement surgery, but for a substantial group of patients who do not respond to the current conservative treatment, this is not (yet) indicated. The RADIOPHENOL study investigates if denervation of knee sensory (genicular) nerves can serve the gap between conservative and invasive treatment for younger patients and for patients who cannot undergo joint replacement surgery due to comorbid health conditions.

The RADIOPHENOL study is a multicentre unblinded randomised controlled trial with three parallel arms (1:1:1). In total, 192 patients with knee OA Kellgren-Lawrence grades 2–4 but not eligible for joint replacement according to the orthopaedic surgeon due to young age, old age and/or comorbidity or technical reasons are eligible and will be randomised to three groups of 64 patients. Group A: traditional radiofrequency ablation, group B: chemical neurolysis with phenol, group C: conservative medical management. Primary outcome is the Oxford Knee Score at 6 months. Secondary outcomes include Western Ontario and McMaster Universities Osteoarthritis Index, knee pain by numeric rating scale, physical functionality, health-related quality of life, mental health, change in medication use, predictive value of a diagnostic block, procedure time, patient discomfort score during the intervention and adverse events.

The protocol (V.2.0, 15 May 2023), was approved by the Ethics Committee of Amsterdam UMC (NL83410.018.22 – METC2022.0890) on 31 July 2023. We aim to publish our results in international peer-reviewed journals.

ClinicalTrials.gov NCT06094660, including the WHO Trial Registration data set items. Registered on 20 October 2023, first patient enrolled on 27 November 2023.

The enteric microbiota drives inflammation in Crohn’s disease. Yet, there are no placebo controlled trials evaluating the efficacy and safety of faecal microbiota transplantation (FMT) in inducing and maintaining remission in patients with active Crohn’s disease. The Microbial Restoration (MIRO) study aims to establish this evidence.

At two specialist inflammatory bowel disease centres, 120 enrolled patients will have a 3-week period of diet optimisation (removal of ultra-processed foods) together with a 7-day course of antibiotics (to facilitate subsequent FMT engraftment). Patients will then be stratified to upper gut (for disease proximal to the splenic flexure) or lower gut (distal to the splenic flexure) disease. Patients will then be randomised in a 2:1 ratio to receive anaerobically prepared stool or placebo for 8 weeks either by gastroscopy, or colonoscopy and enemas. Clinical response at 8 weeks (Crohn’s Disease Activity Index (CDAI) reduction ≥100 points or to 70) receive FMT for weeks 8–16.

Patients achieving clinical response from FMT after 8 or 16 weeks will be randomised in a 1:1 ratio to either a 44-week maintenance phase of FMT or placebo. Patients will receive FMT from one donor throughout the study.

The MIRO study will establish whether FMT is an effective and safe therapy to induce and maintain remission in patients with active Crohn’s disease.

Ethical approval has been received by the St Vincent’s Hospital Melbourne Human Research Ethics Committee (HREC-A 084/21). The results will be disseminated in peer-reviewed journals and presented at international conferences.

ClinicalTrials.gov: NCT04970446; Registered on 20 July 2021.

To assess the evidence for active group-based performing arts interventions for people with Parkinson’s disease (PD).

Scholarly literature (published in English) from any country or countries (last search February 2025). This systematic review was not registered and received no funding.

Five bibliographic databases: AMED (Ebsco), APA PsycINFO (Ovid), CINAHL (Ebsco), EMBASE (Ovid) and MEDLINE (Ovid), plus supplementary searches.

Eligible studies used a quantitative design to assess the benefit of active group-based performing arts interventions on quality of life, functional communication, speech, motor function and cognitive status in PD. The risk of bias was assessed using the SURE, University of York Centre for Reviews and Dissemination and Newcastle-Ottawa Scale checklists. Data were synthesised using narrative synthesis and random-effects meta-analysis.

A total of 94 studies were included: 2453 people with PD (mean age 68 years, 55% male) from 18 countries. Narrative synthesis supported nine combinations of performing arts modalities and outcome domains, including a benefit for dance on motor function (supported by 50 out of 54 studies), dance on quality of life (supported by 24 out of 37 studies) and singing on speech (supported by 17 out of 20 studies). Meta-analysis supported five combinations of performing art modalities, comparators and outcomes, including a clinically significant benefit for PD-specific dance versus usual care PDQ-39, MD –7.81, 95% CI –11.87 to –3.75 and tango-based dance versus usual care on UPDRS-III, MD –9.89, 95% CI –16.65 to –3.13.

Evidence from both the narrative synthesis and the meta-analysis supports a benefit for some combinations of performing arts modalities and outcomes. Limitations of the evidence base included differences in comparators and outcomes, heterogeneity, lack of control arms and male underrepresentation. Future studies should compare the effectiveness of different performing arts modalities, assess functional communication and consider clinical significance.