Special report: new treatments and clinical trials worth knowing about in 2026

Families ask us about new treatments more often than almost anything else. Social media moves faster than the evidence, and Australian access often lags the United States by years. This report, compiled on 2 October 2026, is our attempt to lay out the facts condition by condition with links to the primary sources so you can check for yourself. It is information, not medical advice. Please talk with your paediatrician, neurologist or GP before acting on anything here.

Spinal muscular atrophy (SMA)

SMA is the clearest success story. Newborn screening is now national, with NSW screening since 2022 and an NHMRC-approved national guideline published in April 2025. Babies identified before symptoms can be treated early.

• On the PBS since 1 November 2024: Evrysdi (risdiplam) for adults and pre-symptomatic babies, and Zolgensma gene therapy for pre-symptomatic babies with three SMN2 copies.
• A swallowable risdiplam 5 mg tablet was PBS-listed on 1 April 2026, replacing the refrigerated liquid for older children and adults.
• Higher-dose nusinersen (Spinraza) was approved in Europe in January 2026 and in the US in March 2026. We could not find a TGA approval as of October 2026.

Angelman syndrome

• REVEAL (ION582 / obudanersen, Ionis), Phase 3, NCT06914609: recruiting now at Sydney Children's Hospital Randwick, Queensland Children's Hospital, Austin Health in Melbourne and Perth Children's Hospital, for ages 2 to under 18 and 18 to 50. Primary completion is expected around August 2027.
• GTX-102 / apazunersen (Ultragenyx): the Phase 3 Aspire trial did not meet its primary endpoint, announced 2 September 2026. Australian sites remain in the extension study.

Epilepsy: Dravet and Lennox-Gastaut syndromes

• Fenfluramine (Fintepla) has been on the PBS for Dravet syndrome since 1 May 2025. We could not confirm a PBS listing for Lennox-Gastaut syndrome.
• Zorevunersen (STK-001), an antisense therapy for Dravet, has 162 patients enrolled in its Phase 3 EMPEROR trial with results expected in mid-2027. No Australian site was listed at the time of writing.
• Soticlestat development was stopped by Takeda in January 2025 after Phase 3 failures.

Cerebral palsy

• In April 2025 a five-year-old received Australia's first autologous umbilical cord blood infusion for CP at Monash Children's Hospital, with the case report published in 2026. The MCRI sibling cord blood trial (ACTRN12616000403437) found infusion was safe in 12 children. A 2025 meta-analysis in Pediatrics supports a modest motor benefit. These are early, regulated studies, not a treatment you can buy.
• Cerebral Palsy Alliance's early detection clinics have cut the average age of diagnosis from 19 months to as young as 3 months.
• Recruiting in NSW: CP Movetime (wearable technology, ages 5 to 17, GMFCS IV-V, with Northcott and CPA) and EPIC-CP for Aboriginal and Torres Strait Islander families. See CPA's research studies page.
• Exoskeletons: several are TGA-approved. The NDIS funds therapy sessions that use them but not personal devices; the NDIS Evidence Advisory Committee is reviewing robot-assisted gait training.
• Cerebral Palsy Alliance has a position statement warning against overseas stem cell tourism.

Autism

This is where families need the most care, because the gap between headlines and evidence is widest.

• Leucovorin and paracetamol: in September 2025 the US government announced a paracetamol label change and promoted leucovorin for autism. In March 2026 the US FDA approved leucovorin only for cerebral folate deficiency with a FOLR1 variant and explicitly declined an autism indication. The largest leucovorin-autism trial has been retracted. Australia's TGA said paracetamol remains Pregnancy Category A with no safety investigation open, The Kids Research Institute said the leucovorin evidence "does not come close to the standard needed", and the Australian Academy of Health and Medical Sciences published an evidence brief in 2026 concluding that routine use is not supported. Leucovorin is not TGA-approved for autism.
• Bumetanide (two failed Phase 3 trials), balovaptan (discontinued) and oxytocin (large negative trial) are not treatments for autism.
• Cannabidiol: an Australian crossover trial of 29 children found no change in overall social responsiveness, with some gains in specific behaviours and parent stress. MCRI's trial of CBD for severe behaviour in children with intellectual disability (most of whom are autistic) finished in August 2025 and results are awaited.
• Inklings, an early support program for babies aged 6 to 18 months showing early social communication differences, is running in WA and SA. We could not find an NSW rollout.
• Thriving Kids began on 1 October 2026 and is the main new support pathway for young children. Read our launch explainer.

ADHD

• NSW prescribing reform: since 1 September 2025 GPs can become "continuation prescribers" for stable patients aged 6 and over; 749 GPs had qualified by January 2026. From March 2026 about 300 GPs began training to diagnose and start ADHD medication, with the first finishing in late 2026. This should shorten the long waits for paediatricians and psychiatrists, especially outside metropolitan Sydney.
• Centanafadine (Simtriyo), a new class of ADHD medicine, was approved in the US on 24 July 2026 for ages 6 and over. It is not TGA-approved or PBS-listed.
• Supply of lisdexamfetamine (Vyvanse) has largely stabilised after generics entered the market; the TGA published updated shortage guidance in February 2026.

Rett syndrome

Trofinetide (Daybue) was invented in Melbourne by Neuren Pharmaceuticals, is approved in the US (with a new powder formulation from December 2025), and received European approval in 2026. Neuren's August 2026 filings state it is not approved for sale in Australia, and we found no PBAC submission. Access here would currently be through the TGA Special Access Scheme at the family's or hospital's cost.

Duchenne muscular dystrophy

• Vamorolone, givinostat and Elevidys gene therapy are approved in the US but not registered in Australia. Elevidys has had serious safety restrictions after two deaths in non-ambulatory patients in 2025, and a new global Phase 3 was announced in April 2026.
• Recruiting in Sydney: The Children's Hospital at Westmead is a site for the IMPACT DUCHENNE trial of SGT-003 micro-dystrophin gene therapy (NCT07160634) for ambulant boys.

Fragile X

Zatolmilast missed its primary cognitive endpoint in both Phase 3 trials in May 2026. There is no new approved medicine.

Genomics and undiagnosed disability

• Genomics Australia, a new federal agency, launched in July 2025.
• The Australian Undiagnosed Diseases Network is returning results to families through 2026. Australian Genomics reports that about one in three rare disease participants received a diagnosis, rising to about half in acute care settings.
• Medicare-funded reproductive carrier screening for cystic fibrosis, SMA and fragile X has been available since November 2023.
• In April 2026 the Royal Children's Hospital delivered Australia's first topical gene therapy for epidermolysis bullosa.

Neurotechnology

Synchron's Stentrode brain-computer interface trial (FOCUS-AUS, NCT07533903) began at the Royal Melbourne Hospital in May 2026, recruiting 10 people with severe weakness in both arms. It is a research device, not an NDIS-funded product.

Psychedelic-assisted therapy

Australia's authorised prescriber scheme covers MDMA for PTSD and psilocybin for treatment-resistant depression only. The TGA loosened some requirements in May 2026. There is no approved use for autism, and treatment is out of pocket, typically reported at $15,000 to $30,000. An Australian trial of MDMA-assisted therapy for social anxiety in autistic adults has been planned; we could not confirm its current status.

How the NDIS fits in

The NDIS does not fund medicines, clinical trials or medical treatment; those are the health system's responsibility. What the NDIS can fund is the support around them: therapy to build on gains after treatment, support workers to attend appointments, assistive technology, and capacity building. If a new treatment changes your child's needs, that is a legitimate reason to ask for a plan variation. Talk to us or your support coordinator about the evidence you will need.