Childhood dementia is a devastating condition caused by more than 145 rare genetic disorders, affecting 1 in every 2,900 births.

For children and their families, the impact is profound. As the disease progresses, children lose the skills they’ve worked so hard to develop; walking, talking, eating, and face a rapid decline. With no effective treatments, half of these children will not live to see their 10th birthday, and 70% will not reach adulthood.

But there is hope on the horizon.

A promising drug, N-acetyl-L-leucine (NAL), has already been approved in the United States for one form of childhood dementia, Niemann-Pick disease type C. It is also being trialed for other conditions, and early laboratory research here in Australia suggests that NAL could benefit other forms of childhood dementia that currently have no approved treatments. These include Sanfilippo syndrome, which robs children of their abilities and often leads to death in their teenage years, and GM1 gangliosidosis, an even more aggressive condition where some children diagnosed in infancy may not survive beyond their second birthday.

For families, these diagnoses are heartbreaking, and the lack of treatment options compounds their despair. That’s why the WCH Foundation is proud to support Professor Kim Hemsley’s groundbreaking research to evaluate NAL as a potential treatment for these devastating diseases.

This work will also lay the groundwork for clinical trials, bringing us closer to life-changing therapies for these devastating conditions.

“Wins are few and far between in medical research, and for us in this field, it’s personal. We know these families, and we care about these families. So to realise that you have the funding to potentially make a difference, that’s a very good day.” – Professor Kim Hemsley

Through this vital research, Prof. Hemsley and her team are working to transform the future for children living with childhood dementia. By building on promising laboratory findings, this project has the potential to bring life-changing treatments within reach for children and families affected by this devastating disease.

Find out more about the research we support, here.

Every year, one in seven babies are born with seizures, developmental delays, or the risk of early death due to genetic changes that disrupt brain development.

Despite advances in identifying the genes involved, most of these children face a lifetime without effective treatments, leaving families with heartbreak and uncertainty.

A breakthrough technology called antisense oligonucleotides (ASOs) is offering new hope. These genetic therapies, already proven safe and effective in other diseases, have the potential to transform outcomes for children with rare and devastating brain disorders. By targeting the root cause of these conditions at the genetic level, ASOs could provide treatments where none currently exist.

At present, children with conditions like PCDH19 clustering epilepsy, a severe form of epilepsy that primarily affects girls, and TIMMDC1 neurodegenerative disorder, a rare and life-limiting disease, have few, if any, treatment options. Families are left with no therapies that can truly change their child’s future, compounding the distress of these diagnoses.

The WCH Foundation is proud to support Professors Jozef Gecz and Paul Thomas, along with their multidisciplinary team, in their groundbreaking research to develop ASO therapies for these conditions.

Their work will test new ASO therapies in models that reflect how these diseases affect children, identify safe and effective doses to give children the best possible chance of benefit, and build a treatment pipeline that could be applied to many other rare childhood brain disorders in the future. This research represents a critical step forward in the fight against rare genetic brain disorders, offering families the possibility of treatments that were once unimaginable.

“The donors who make this possible are just phenomenal. I’m eternally grateful to everyone who donates to health and medical research, because in one way or the other, it comes back to all of us.” – Professor Jozef Gecz

Find out more about the research we support, here.

Helping infants feel safe, supported, and understood is one of the most powerful ways parents can nurture their child’s mental health and set the stage for positive social and emotional development.

Yet, for many new parents, the transition to parenthood is filled with the challenges of sleepless nights, soothing a crying baby, and building a strong parent–infant bond. Learning how to navigate these challenges before birth can make a world of difference, but few parents are given this opportunity.

To address this gap, Dr Alyssa Sawyer and her team at the University of Adelaide, SA Medical Imaging (SAMI), and the Women’s and Children’s Health Network (WCHN) developed an attachment-focused intervention. A pilot trial showed that parents facing social and emotional adversity improved their ability to bond with their baby, felt more confident as parents, and gained valuable knowledge about safe infant care. Importantly, parents who participated in the trial asked for one key change: to make this support available to all new parents, not just those at risk.

The WCH Foundation is proud to support the development of this program as a universal intervention, ensuring every family can benefit. The research team will work with parents and clinicians to adapt the program for delivery alongside routine care.

“What we’re hoping we’ll achieve is designing this intervention in a way that can support all parents who come through the hospital to have support for psychological and attachment relational health supported around their routine care across the antenatal and postnatal period.” – Dr Alyssa Sawyer

The program combines innovative approaches to support parents before and after birth, including attachment-focused ultrasounds at the 20-week scan to help parents connect with their baby, antenatal virtual home visits during pregnancy to help parents reflect on their baby’s experiences, and postnatal virtual home visits to provide guidance on bonding, managing crying and sleep, and supporting emotional development.

If successful, this project could strengthen parent–infant bonds, build parents’ confidence, promote infant mental health, and ensure universal access to early support, giving every baby the best possible start in life.

Find out more about the research we support, here.

Borderline Personality Disorder (BPD) is a serious mental health condition that makes managing emotions incredibly challenging. For mothers with BPD, who often carry their own histories of trauma, this can make it harder to respond to their baby’s needs and build the secure attachment every child relies on for healthy development.

When this bond is disrupted, children face a greater risk of developmental, emotional, and mental health challenges later in life. For mothers, the experience can be overwhelming, often leading to feelings of guilt and failure that make parenting even more difficult.

The Mother-Infant Dialectical Behaviour Therapy (MI-DBT) program, developed by the Women’s and Children’s Health Network (WCHN), is changing lives. This 24-session group program teaches emotional regulation and parenting skills to help mothers strengthen their bond with their babies. Mothers who participate often show incredible determination to give their child the best start in life, and the program has been highly successful in improving mother-infant relationships.

However, demand for MI-DBT far exceeds availability. Families face long waits for a place, and for those in rural or remote areas, the program is often out of reach entirely.

The WCH Foundation is proud to support A/Prof Anne Sved Williams and her team (Dr Rebecca Hill, pictured) in developing a telehealth version of MI-DBT, making it accessible to families no matter where they live.

“The benefit is in the relief of both their distress but also changing the trajectories for those kids so they might go on to have better futures and in turn, not repeat that intergenerational transmission.” – Dr Rebecca Hill

This innovative project will expand access to MI-DBT for mothers in rural and remote communities, reduce wait times for families eager to get help, and train more clinicians to ensure the program can grow and be delivered consistently nationwide. By breaking down barriers to care, this project has the potential to transform the lives of mothers and their children, building stronger, healthier families.

Find out more about the research we support, here.

The Women’s & Children’s Hospital Foundation is proud to announce its 2025 research grant recipients, leading a diverse array of groundbreaking projects aimed at improving health outcomes for children and families.

Almost $3 million has been committed to these projects over the coming years, supporting vital research into mental health and rare diseases:

• Associate Professor Anne Sved Williams and her team will develop and evaluate telehealth delivery of a program for mothers with Borderline Personality Disorder, enabling them to access mental health support from home, no matter where they live. 

• Dr Alyssa Sawyer and her team will evaluate an attachment-informed perinatal care program designed to strengthen parent–infant bonds, build parental confidence, and promote lifelong wellbeing. 

• Professor Michael Stark and his team will trial late amino acid administration in infants requiring urgent surgery for serious and rare abdominal defects through the DELA3Y Trial, to improve recovery outcomes for these vulnerable babies. 

• Professor Jozef Gecz and his team will develop oligonucleotide therapies for two rare disorders causing epilepsy. This groundbreaking research could not only provide real treatment options for these rare disorders but also open the door to transformative therapies for many others. 

• Professor Kim Hemsley and her team will conduct pre-clinical evaluation of a potential drug treatment for childhood dementia, offering a beacon of hope for children and families affected by this devastating condition.  

• Dr Karissa Barthelson and her team will use zebrafish models to investigate skeletal phenotypes in Morquio syndrome, to uncover new insights into the condition, paving the way for improved treatments. 

These projects aim to create transformational change in healthcare for families across South Australia and beyond.