Investigating the impact of music for children with Batten disease

Abstract

Batten disease is a rare neurodegenerative condition affecting approximately 150–200 children and young people in the United Kingdom. It leads to progressive loss of skills, with most individuals not surviving beyond adolescence. While current research primarily focuses on clinical trials for gene and enzyme replacement therapies, there remains an urgent need for therapeutic and educational strategies that support affected children and their families in the present. Music has been identified as a meaningful support mechanism for individuals with Batten disease, yet empirical research in this area is limited. Drawing on developments in neuroscience and music therapy, this study explores how music supports children with Batten disease through semi-structured interviews with parents and carers. Thematic analysis revealed three key themes: music and well-being; music’s role in supporting functional skills; and the use of music in care settings. Findings highlight music’s emotional, communicative, and functional value, providing insights for educators and healthcare professionals. This research contributes to the growing evidence base for music-based interventions for rare neurodegenerative conditions.

Background

Batten disease, the most common form of the Neuronal Ceroid Lipofuscinoses (NCLs), is a rare but devastating group of inherited lysosomal storage disorders that primarily affect children. These conditions lead to progressive neurodegeneration due to the accumulation of cellular waste in the brain, resulting in a steady decline in physical, cognitive, and sensory functioning (Mole and Cotman, 2015).

While medical research has advanced to the point of offering one gene therapy (cerliponase alfa) for a single variant (CLN2), most children affected by Batten disease have no disease-modifying treatments available (Lewis et al., 2019). Clinical management focuses primarily on symptom control through pharmacological interventions, seizure management, physiotherapy, and palliative care (Augustine et al., 2013). However, these interventions offer limited support in preserving quality of life or meaningful interaction as the disease progresses.

Given these limitations, families often turn to alternative, non-pharmacological strategies to support their children’s well-being. Among these, music has emerged as a powerful and accessible tool. Preliminary studies suggest that music can offer moments of emotional clarity, connection, and calm for children with Batten disease, while also helping to stimulate cognitive, communicative, and physical responses (Atkinson, 2022; Elmerskog et al., 2019; Ockelford et al., 2019; von Tetzchner et al., 2013, 2019). Research from related neurological conditions, such as Parkinson’s, Rett’s syndrome, and traumatic brain injury, have further shown how music-based interventions can help maintain motor skills, improve speech pacing and clarity, and enhance cognitive awareness (Harris et al., 2016; Mainka et al., 2018; Thaut, 2014).

Findings from the author’s doctoral work, highlight music’s unique potential not only to enhance children’s functional abilities but also to contribute to broader emotional and relational well-being, creating rare moments of recognition, engagement, and joy amid progressive loss (Atkinson, 2022). For children who are gradually losing their ability to communicate, move, and interact, music may serve as one of the last remaining pathways for connection (Atkinson, 2025). Yet, despite this growing interest, there is little research exploring how parents and caregivers themselves perceive the impact of music in daily life.

This study aims to address that gap by exploring the experiences of caregivers who support a child with Batten disease, focusing on their perceptions of how music supports their child’s well-being and functional skills. By grounding the research in caregiver insights, this study contributes to the growing call for holistic, arts-based approaches to care that prioritise quality of life alongside clinical management.

Aim

This research aimed to explore the role and perceived importance of musical experiences for children affected by Batten disease, as reported by parents and carers. Through interviews, participants reflected on how music is used within the home environment while supporting a child with Batten disease, and the ways in which these musical interactions may contribute to the child’s well-being and daily functioning.

Methods

In 2024, The Musicians Company funded a project exploring the importance of music for children with Batten disease. As part of this project parents provided interviews in relation to their musical experiences of supporting a child with Batten disease at home. Data represented interviews with parents of a child with Batten disease or those who cared for children affected by Batten disease.

Participants

All participants were recruited through the parent advocacy charity Batten Disease Family Association (BDFA) in 2024. The BDFA disseminated a research poster on behalf of the researcher through a mailout and a research poster on their social media channel.

Inclusion Criteria were as follows:

• A parent or carer supporting a child or individual with Batten disease.

• The child could be alive or deceased.

• The participant must be over the age of 18.

• The child may have any of the 14 variants of Batten disease.

• Parents or carers who support multiple children with Batten disease.

• Exclusion Criteria:

• Anyone who is not directly supporting a child with Batten disease.

• Any parents or professionals under the age of 18.

• An inability to complete an interview in English.

Four parents (three mothers and a father) and one carer of two children affected by Batten disease (two of three triplets) agreed to take part in an interview. All interviewees supported an individual with Batten disease at home with ages ranging from 5 to 7 years of age and one individual who is 45 years of age (see Table 1).

Table 1.

Demographic information of participants and their children.

Participant	No. of Child(ren)	Child’s Age	Diagnosis	Relationship to child
PC001	1	5	CLN2	Mother
PC002	1	45	Adult Onset	Mother
PC003	1	45	Adult Onset	Father
PC004	1	5	CLN1	Mother
PC005	2	7	CLN2	Carer

Ethical approval was obtained from the Anglia Ruskin University Ethics Board in July 2024.

Data collection

All interviews were conducted remotely by video call between November 2024 and March 2025, and each participant took part in a one-on-one (or two-on-one with a couple) interview with the researcher. Interviews lasted between 25 minutes and 1 hour and were recorded using the in-app Teams or Zoom recording function.

All participants had no prior relationship to the researcher beforehand; therefore, interviews all opened with an overview of the research project, its aims, interests, and the interviewer’s background as a Music Therapist and researcher. The content of the interviews focused on a reflection of their experiences supporting a child at home. All participants were asked about their experiences of music in or around the home, and the children referenced in interviews were not receiving music therapy sessions.

A full interview schedule can be found in Appendix 1. The interviews were semi-structured allowing for a rich discussion between the interviewer and the interviewee, ensuring the flexibility for topics to emerge, reflected upon and discussed (Fletcher, 2017).

Analysis

Interview audio files were transcribed by the first author, using MS Teams, Zoom and Microsoft word. The data were mapped onto the pre-existing codes and themes by the first author with support from a research intern using a deductive thematic analysis approach, as outlined by Braun and Clarke (Braun & Clarke, 2006).

Rather than developing themes inductively from the data, the analysis was guided by a pre-existing thematic framework developed during earlier doctoral research by the first author (Atkinson, 2022), which explored the perceived impact of music from the perspectives of professionals working with children with complex needs.

The decision to use this deductive framework was twofold. First, the original framework had already been robustly developed through extensive data collection and analysis with professional’s experiences in music for children with neurodegenerative conditions, making it a suitable foundation for further exploration. Second, the use of a pre-existing framework allowed for systematic comparison across participant groups, in this case, between parents and professionals, regarding shared or differing perceptions of music’s role and impact. The data were therefore mapped onto the identical themes and subthemes from the previous thematic analysis (Atkinson, 2022).

By applying the same thematic structure to the parent data, the analysis aimed to identify areas of convergence and divergence in how these two key stakeholder groups conceptualise the benefits and challenges of music. While the overarching themes remained consistent, nuanced differences emerged in emphasis, language, and priority. These similarities and differences are highlighted in the discussion section, with particular attention paid to how parental insights either reinforce or extend the professional perspectives underpinning the original framework. Where relevant, subthemes were expanded or refined to reflect novel insights from parents’ or professionals’ lived experiences.

Ethical considerations

The best interests of the participants were a fundamental concern for the research team, particularly psychologically. The interviews with parents and carers were focused on their role supporting a child with an incurable disease, which they may have found distressing. To manage this risk, the interviewer was the first author, who has conducted research like this previously and has 8 years’ experience working with parents and staff from this highly fragile community. The interviewer is also a registered Music Therapist with HCPC and has training in working with sensitive topics or populations. Under the HCPC regulations, the interviewer kept all information confidential, anonymised and was mindful of the parent or carer’s well-being at all points in the research. The participants were made aware that they had the right to withdraw at any time.

Results

This deductive thematic analysis explores the diverse and profound ways music influences the lives of children and young people with Batten disease. Across multiple themes and subthemes, the qualitative data reflects the impact of music on communication, emotional well-being, physical expression, cognition, and daily routines (see Figure 1). Results demonstrate how music emerges not only as a tool to support care but also as a medium of connection, identity, and joy. Themes here outline the overall sentiment that parents and carers understood the importance of music and music therapy considering how both could be used to support the child now and in the future.

Figure 1.

Thematic map derived from parent and carer interview data (Atkinson, 2022).

Music and well-being

Parents and carers fundamentally outlined how music plays a profound role in supporting the emotional and psychological well-being of children and young people with Batten disease. They highlighted how music often evoked spontaneous, visible reactions, even among those with significant physical limitations, offering moments of connection and joy.

One caregiver described, ‘We’d take her to the piano and move her fingers for her . . . that would lead to eye movements and recognition’ (PC002), illustrating how even passive engagement with music can trigger meaningful responses. Another parent vividly recalled, ‘You take his hands . . . it’s like the sun coming out from behind a cloud . . .[music] reaches him, and it brings him out of himself’ (PC004), describing a transformation in affect and awareness when music and physical interaction are combined. These instances reflected how music could break through cognitive and sensory impairments to foster recognition, well-being, and quality of life.

Beyond immediate responses, parents also spoke of how music provided grounding and emotional security. Familiar songs and voices, whether from caregivers or recordings, helped maintain a sense of identity and continuity. As one caregiver put it, ‘She connects in some sort of way with that song . . . you can just see all the emotion on her face’ (PC005). Many described how music becomes an auditory anchor in a world that may otherwise feel fragmented or confusing, especially for those experiencing visual impairment or cognitive decline:

I think that once you’ve lost visual cues and the light and dark circadian cues, I can’t imagine that it’s easy to keep a grip on reality if you’re just moved around silently going through life. So, I do think you need to give someone anchoring, you know, and for him, you know, we sort of landed on the songs that worked. (PC004)

Moreover, music’s capacity to regulate emotion was consistently highlighted. Specific songs often sooth agitation and anxiety: ‘The only thing that would calm him down was very specific songs . . . it literally had to be Amazing Grace’ (PC004) one parent shared. Many shared how in moments of upset or overstimulation, music offers a consistent and reliable pathway to calm.

Parents also spoke of how music supported emotional expression, even when verbal language is declining or lost: ‘the fact that she’s got sounds . . . or the music that she used to play and love and sing, I think that’s vitally important, really’ (PC003) said a parent of a child responding to a familiar tune. The musical experiences offered a channel for emotional expression that may otherwise remain locked away.

This effect extended to boosting mood and enhancing confidence. One parent recounted how a musical toy reawakened joy or playfulness: ‘that Caterpillar has been life-changing for us . . . it brought him back to play with us’ (PC004). Others reflected on how children who may struggle to engage with the world in traditional ways, find in music a lifeline that nurtures confidence and well-being. One parent reflected, ‘She got up and sang . . . She did enjoy singing and music. There’s no doubt about it. It was a passion in her life’ (PC003) affirming the lasting impact of music on self-esteem and self-expression.

Enhancing functional skills

Parents also described the way in which music enhances a variety of functional skills, particularly communication, language, memory, and movement. Specifically, it facilitated social interaction in ways that are often otherwise inaccessible. As one parent explained, ‘He’s engaging with me and we’re having a sort of communication and he’s happy, and then if you stop, he might tense for more. So, he’s actively participating in his life then in a way that very little else can help him do’ (PC004).

Parents recounted how music offers a shared language, a means of mutual recognition and enjoyment that bypasses conventional verbal exchange. It also acted as a stimulant for speech and vocabulary. Other parents reflected on how music supported physical speech, ‘when the [music] comes and I look in the mirror and I can see a face beam and then halfway through the song, she’ll chirp in and say “Moana”’ (PC001).

Environmental sounds, such as bird songs, also remain particularly important for children with Batten disease. ‘He responds a lot to birds tweeting in the trees’ (PC004) one parent noted, emphasising how sound helps children understand and interact with their environment. Others reflected on the importance of sound ‘. . . all the other sensors are going, but as hearing isn’t. So perhaps that’s where you can say that music has a greater contribution’ (PC003).

Parents spoke of how routine was also significantly supported by musical cues. Families consistently used songs to mark transitions and structure the day. One parent explained,

We had specific songs for nappy changes, songs at bedtime, cleaning his face in his music chair . . . He had the little snooze cloud that plays a certain tune, when we lie him down to sleep. So, he has various auditory cues that let him know what’s happening as part of his routine and also to support his well-being. (PC004)

These auditory anchors helped children anticipate and understand daily routines, offering predictability and comfort. Others reflected on the consistency and familiarity with songs and routines that could provide crucial structure, ‘the consistency of routine, words, songs . . . gives him a structure he understands’ (PC004) another shared. This routine when supported by music not only enhanced well-being but also reduced anxiety and confusion. For one adult with Batten disease, her parents also spoke of the importance of music as a routine: ‘[Music] is always there and that kind of routine I think is linked to the music. So, the routine and the music kind of go together’ (PC002).

The cognitive benefits of music were also evident in both memory retention and learning. Parents spoke of how children often remember childhood songs even after other skills decline. ‘She’d recognise a song and in the early stages sing along to it’ (PC003) one parent shared, while another explained,

A couple of the childhood songs remain, and he became obsessed with one of them. It was his favourite thing in the world . . . So if he was ever distressed at night, you literally would sing that, or if worst came to worst, play him a little bit of the YouTube video and what would be calm again, it was they were literal silver bullets for everything, these songs. (PC004)

Another described how ‘Music helps people with these type of conditions, to recall good memories and to have flashbacks ‘That bit of music . . . I was in such and such a place when I last heard that’ (PC002) said one parent, describing how a simple tune evoked powerful associations.

Music not only tapped into long-term memory but could also be used to introduce new information or reinforce recent learning in conjunction with speech: ‘It’s so important for the memory, the speech, because even if she doesn’t want to talk, she’ll sing a song and she’ll remember the song’ (PC001). Parents and carers expressed how familiar songs helped maintain memories, but also new songs can become part of a child’s cognitive learning.

In addition, parents spoke of how music supported physical skills, particularly movement. Parents recounted how rhythmic engagement often prompted movement even when mobility was limited. As one caregiver described, ‘She was obsessed with dancing . . . just bending at the knees and up and down’ (PC005). Another remarked, ‘clapping to music is basically the mainstay of him being able to show pleasure’ (PC004) emphasising the link between music and physical skills. For other children, regular music and movement groups helps maintain physical skills, ‘. . . she goes to this group every Saturday and she loves it, loves the dancing, and it’s keeping the mobility up and her strength and her core muscle’ (PC005).

They also spoke of how music could help with muscle memory and movement recalling how:

The music memory unlocked her and she were moving her hands and her legs and knowing what to do when. It was really odd. It was really strange, but that’s a learned skill from the music class and she just went into the full thing. (PC001)

Other parents talked of how music helped to maintain physical skills:

. . . as with everything . . . it’s use it or lose it. So, engagement and excitement and a getting him to have a physical response is, is important. Getting him to still be able to smile and laugh . . . I think what was important to me is, as his skills fade, to not just have him end up sitting left alone and unstimulated in a corner because he can’t do things now. (PC004)

Music in care

Importantly, parents and carers discussed how music played a role in caregiving. This was multi-faceted; providing both practical and emotional support in managing progressive challenges associated with Batten disease. As motor, cognitive, and visual abilities decline, parents expressed how music often remained accessible as a distraction or diversion. One parent said ‘. . . we pulled the string and a particular tinkling tune came out that would always make him smile and laugh and tolerate nappy changes’ (PC004) describing how they used musical toys to support personal care.

Caregivers often discussed their strategies to care and how they creatively adapted sounds and music to maintain interest and connection for the children in their care. ‘I just kept changing things . . . silly voice, high, low . . . to create a random word or a random sound’ (PC005) one carer said, demonstrating how innovation keeps musical interactions engaging and accessible.

Multisensory approaches were also crucial for some children. Many families incorporated toys that combined sound and touch – such as music boxes or jingly stuffed animals – to sustain attention and evoke reactions. One child, for instance, ‘would spend hours just making [a musical toy] boing and waiting for the music’ (PC004). Others spoke of how music could provide distraction:

I sang song after song after song after song, and it was funny because the minute I did stop singing, you know, she would get stressed. So, it’s it just has become very apparent to me that it’s just totally needed. (PC005)

Finally, parents spoke of how music also played a role in creating memories and fostering joyful experiences. They spoke about how music acted as a bridge to the past and a spark for new memories. Others reflected on the impact of musical experiences:

[music] it gave her lots of experiences, that I certainly couldn’t have done. Music contribution throughout her life has been good . . . from singing in the choir scene . . . She saw Michael Jackson. She’s seen Bon Jovi. You know, so she has seen some big places at big concerts. (PC002)

One parent summarised the experience by simply stating that ‘Music is vitally important to the well-being of these people, really . . . it has a massive role to play’ (PC003). Another expressed how ‘music and sounds in general provide a gateway into the world . . . of interacting with the world and understanding the world’ (PC004).

Discussion

Findings from the data support previous literature alluding to the impact of music on well-being, functional skills, such as communication, physical movement and cognitive awareness and music for care (von Tetzchner et al., 2013).

Specifically, supported by recent research in the field, parents and carers describe the unique effect that music had on creating a well-being experience for children, where there were moments of clarity and connection (Atkinson, 2022). With a focus on quality of life and well-being, data demonstrate the importance of using arts-based activities such as music as a tool for helping improve well-being, facilitate calm and improve mood.

This aligned with data from interviews with professionals, where school staff described how music could offer a tool to enhance emotional well-being, and bring moments of clarity for children with Batten disease attending school (Atkinson, 2022). Specifically, during moments of anxiety or confusion in the school environment, professionals agreed that music could invoke calm and offer grounding, particularly in moments of disorientation. They also concurred that music was a useful tool to create auditory markers, helping children to use sounds to navigate through routine and activities in their school day.

Comments from parents and carers clearly described the unique way in which music could preserve skills for the child, such as mobility, cognitive skills, and communication. Akin to the themes derived from previous research, professionals similarly described the way in which activities like singing and dancing could enhance and maintain functional skills like speaking, moving, self-expression and understanding (Atkinson, 2022). Comparing previous research in the music therapy sector, data aligns with research for other populations where music has an impact on physical skills and improving mobility (Mainka et al., 2018), orientation and awareness (von Tetzchner et al., 2019), and also speech pacing and clarity (Harris et al., 2016).

From previous research, interviews with professionals expanded upon this theme of preserving skills, commenting on the use of music to support inclusivity in a school environment (Atkinson, 2022). Here, professionals highlighted the importance of music to close the widening gap between children with Batten disease and their peers, commenting on how music could be universally understood and enjoyed by children and their peers inclusively.

There were, however, limitations in the current research study. First, the small sample size challenges the ability to make generalisations from the findings from this article. The research accepts the limitations of the small sample case study design which was largely dictated by the small population of children with Batten disease in the United Kingdom, and the small pool of parents who were able to make the time to give an interview.

Second, recruitment of participants was also conducted through convenience sampling and therefore may not have represented of the wider perspectives of other parents and caregivers in the United Kingdom. Parents and carers in this study may have been interested in participating because their child or children has a particular affinity to music. The research therefore acknowledges the exploratory nature of this project with future research aiming to increase the sample size to gather a larger representative pool of participants for the population of individuals with Batten disease and their supporting individuals.

Conclusion

Experiences here illustrate how parents and carers described a fondness and appreciation of music, and many explain how they utilise music in many aspects of their role to support well-being and happiness. Parents and carers reflected on the positive impact that music had on preserving functional skills, such as speech and communication, orientation movement and cognition. Many suggested that music and rhythm could help support the child to communicate independently and effectively with those around them, recommending that music could help more children with Batten disease.

While these musical experiences are often informal and unstructured, their reported impact aligns closely with established music therapy principles. Findings from this research suggest that while parents and carers observe meaningful benefits from everyday musical experiences, these impacts could be further amplified if music were delivered in a structured, intentional, as goal-directed music therapy. Part of the author’s doctoral research (Atkinson, 2022) also provided an in-depth exploration of music therapy practice with children affected by Batten disease. Results highlighted that long-term music therapy could support emotional regulation, maintain interaction and communication, and preserve a sense of identity as cognitive and physical abilities decline.

Evidence from broader paediatric neurodisability research further supports the need to investigate music therapy’s potential role in Batten disease. Bringas et al. (2015) demonstrated that structured music therapy interventions can support neurorestoration in children with severe neurological disorders, including improvements in attention, motor coordination, and emotional responsiveness. Although Batten disease is degenerative rather than static or acquired, these findings suggest that music-based interventions may help optimise remaining neurological function, slow functional decline, or enhance quality of life by engaging preserved neural pathways.

Data in this article importantly begin a dialogue between perceptions around the impact of music, and the unheard, lived experiences of those supporting children affected by Batten disease. Findings situate the project within a wider arena of research investigating the potential of music therapy interventions and programmes for those affected by neurodegenerative disease.

Together, these findings highlight a critical gap between the observed benefits of music in daily life and the limited evidence for music therapy as a structured intervention for children with Batten disease. While families and professionals have reported meaningful engagement through music, there remains a need for further research examining how music therapy can be systematically implemented, evaluated, and integrated into multidisciplinary care. Understanding this distinction is essential for developing evidence-based, holistic support strategies that prioritise both functional ability and relational well-being.

Building on this foundation, ongoing research would involve a larger-scale mixed-methods study. The next stage of research will explore the longer-term impact of music therapy interventions across a broader range of child populations affected by rare and complex diseases, with the aim of deepening understanding as to the physiological impact of music interventions for children with complex and rare conditions.

Ongoing collaboration remains central to these research efforts. Continued partnerships with the Batten Disease Family Association, charitable trusts, and the broader music therapy community will support advocacy for improved access to services, enhanced training opportunities, and necessary policy changes to ensure equitable support for affected families.

Supplemental Material

sj-docx-1-bjm-10.1177_13594575261423388 – Supplemental material for Investigating the impact of music for children with Batten disease

Supplemental material, sj-docx-1-bjm-10.1177_13594575261423388 for Investigating the impact of music for children with Batten disease by Rebecca Atkinson in British Journal of Music Therapy