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Patients & Families | Dawn Therapeutics

Patients & Families

Support for Patients and Families

This page is designed for families who want clear, respectful information about Hurler syndrome (MPS I-H), current care, and how to find trusted support. We also offer a Dawn Therapeutics Patient and Family Information Pack to help you organise appointments, questions, and next steps.

Important

Dawn Therapeutics is a preclinical company. We do not have clinical trials open at the moment. This page is for education and support, and does not replace advice from your child’s specialist team.

Clear family-friendly information No current clinical trial recruitment Patient & Family Information Pack available Links to trusted sister-site resources

Request information pack Browse HurlerSyndrome.org resources

At a glance

✓Hurler syndrome is the severe form of MPS I and affects multiple organs over time.
✓Early diagnosis matters because treatment planning is time-sensitive.
✓Current care is built around HSCT, ERT, and long-term supportive multidisciplinary care.
✓Families often need support for hospital care, education, mobility, and mental wellbeing.

What this page can help with

✓Understanding the condition in simple language
✓Knowing what questions to ask your care team
✓Finding trusted resources for families
✓Requesting a free information pack from Dawn

Start here Understanding Hurler syndrome Simple overview for families Next steps Diagnosis and care pathway What usually happens next Treatment Current treatment and care HSCT, ERT, supportive care Support Request family information pack Send a request to the Dawn team

Understanding Hurler syndrome in simple terms

Hurler syndrome, also called MPS I-H, is a rare inherited condition. Children with Hurler syndrome have very low or absent activity of an enzyme called alpha-L-iduronidase (IDUA). Without enough IDUA, natural substances called glycosaminoglycans (GAGs) build up inside cells and gradually affect many parts of the body, including the bones, heart, lungs, and brain.

Cause

IDUA enzyme deficiency due to changes in the IDUA gene.

Inheritance

Inherited in an autosomal recessive pattern.

Pattern

Progressive condition that affects multiple organ systems.

Early signs that often lead to diagnosis

Symptoms usually start in infancy and may become more obvious over the first few years of life. Families often notice a pattern rather than one single symptom.

Infancy

Hernias, frequent infections, mild developmental delay, and early facial changes.

Toddler years

Joint stiffness, reduced mobility, enlarged abdomen, snoring or noisy breathing.

Why timing matters

Early referral helps the specialist team plan treatment more quickly.

Diagnosis and next steps

What usually happens after suspicion or screening

If Hurler syndrome is suspected, or if newborn screening is positive for MPS I, families are usually referred urgently for confirmatory testing and specialist review. A positive newborn screening result is not a diagnosis by itself, and more tests are needed.

Confirmatory tests

IDUA enzyme testing, urine GAGs, and IDUA gene analysis are commonly used to confirm the diagnosis.

Metabolic specialist referral

A specialist metabolic team coordinates assessment and helps explain the diagnosis and treatment pathway.

Treatment planning

The team discusses treatment timing, transplant suitability, enzyme therapy, and urgent supportive care needs.

Family support and care coordination

Families are usually supported through hospital visits, practical planning, and links to trusted support organisations.

Questions you can ask your specialist team

It is completely normal to feel overwhelmed. Bringing a written list of questions can help during appointments.

Diagnosis and severity

What tests confirmed the diagnosis, and what do they show about severity and urgency?

Treatment options now

Is HSCT being considered, is ERT needed now, and what is the expected timeline?

Specialist monitoring

Which specialists will be involved (heart, lungs, orthopaedics, ENT, hearing, eyes, dental, neurodevelopment)?

Emergency plan

Who should we contact if breathing, surgery, anaesthesia, or sudden health issues arise?

Tip: Keep a folder (paper or digital) with letters, scan results, medicines, and clinic summaries. It makes appointments and emergencies much easier.

Current treatment and care

The three pillars of current care

Current care for severe MPS I-H is built around three pillars: haematopoietic stem cell transplantation (HSCT), enzyme replacement therapy (ERT with laronidase), and long-term multidisciplinary supportive care. Families often need all three parts of the care system, even if one treatment is the main focus.

HSCT

Stem cell transplant

HSCT aims to provide a long-term source of enzyme-producing cells and is a key disease-modifying treatment for many children with severe MPS I-H.

Most time-sensitive part of the pathway
Best outcomes when done early
Can help protect brain function
Does not fully remove long-term care needs

ERT

Enzyme replacement therapy

ERT with laronidase is given as regular intravenous infusions and can help many body organs and tissues by reducing GAG storage.

Often used before transplant and in ongoing care
Can improve many somatic symptoms
Does not adequately treat the brain
Works as part of a wider care plan

Supportive care

Multidisciplinary long-term care

Because Hurler syndrome affects many organs, supportive care remains essential before and after HSCT or ERT.

Heart, lung, airway, hearing and vision care
Orthopaedic and mobility support
Dental, nutrition and growth support
Developmental and school planning

Living with Hurler syndrome

Support for everyday life

Hurler syndrome affects the whole family. Alongside hospital care, many families need help with education, practical routines, emotional support, and coordination between different teams. Building a support network can reduce isolation and make day to day life more manageable.

Hospital and appointments

Ask for a named point of contact, keep appointment notes, and bring a written question list each time.

Education and school planning

Early school support planning can help with learning, mobility, communication, and attendance around treatment.

Mental health and family wellbeing

Parents, siblings, and carers may all need emotional support. Peer groups and counselling can help.

Speech, hearing, and vision

Regular reviews can support communication, learning, and social confidence over time.

Mobility and daily adaptations

Joint stiffness and skeletal issues can affect comfort and independence, so practical adaptations matter.

Transition and long-term planning

As children grow, families may need support planning for adult services and long-term follow-up.

Family FAQ

Is a positive newborn screening result the same as a diagnosis? +

No. Newborn screening is an early test, not a full diagnosis. If the result is positive for MPS I, the baby will need confirmatory testing such as enzyme testing, urine GAGs, and IDUA gene analysis.

Why does early diagnosis matter so much? +

Hurler syndrome is progressive, so timing matters. Early specialist referral helps the team assess treatment options quickly, especially when HSCT is being considered.

Does treatment end after HSCT? +

No. HSCT is a major treatment step, but long-term supportive care usually continues. Children may still need regular monitoring and support for heart, lungs, bones, hearing, vision, development, and daily living needs.

Can Dawn Therapeutics offer treatment right now? +

No. Dawn Therapeutics is currently preclinical and does not have clinical trials open at the moment. This page is intended to support families with information and signposting.

What is the Dawn Patient and Family Information Pack? +

It is an educational support pack for families that can include a simple overview of Hurler syndrome, a care pathway checklist, appointment question prompts, and links to trusted resources. It does not replace medical advice.

Patient & Family Information Pack

Request a free information pack

If you are a parent, family member, or carer, you can request a Dawn Patient and Family Information Pack. We can send a practical pack to help you organise information, prepare for appointments, and find trusted resources.

First name Last name

Email address Country

I am a... Stage (if known)

Preferred language Child age (optional)

What would help most right now? (optional) I consent to Dawn Therapeutics contacting me about this information pack request. I understand this is educational support and not medical advice. I understand personal details will only be used to respond to my request and support this enquiry.

Integration note: this form is ready for Elementor or your preferred form system. Set the submit action to your handler and keep the thank-you redirect as /patients-families-thank-you/.

Browse resources first

What is included in the pack

We can provide a simple, family-friendly pack to help you prepare for the next stage of care and find trusted information.

✓Plain-language overview of Hurler syndrome (MPS I-H)
✓Diagnosis and care pathway checklist for appointments
✓Questions to ask your specialist team
✓Overview of HSCT, ERT, and supportive care
✓Practical support and community signposting
✓Links to detailed resources on HurlerSyndrome.org

If your child has urgent symptoms, breathing concerns, or is due for surgery or anaesthesia, please contact your specialist medical team or emergency services immediately.

You can also adapt this panel into a downloadable PDF section later if you want a direct "Download starter guide" option.

Trusted resources

Useful pages from our sister site, HurlerSyndrome.org

For detailed educational content, visit HurlerSyndrome.org. It provides patient and family focused information on diagnosis, treatment, supportive care, and living with Hurler syndrome.

Overview Hurler syndrome home page Trusted, science-based information and support for families and carers. Symptoms Symptoms and diagnosis Typical symptom pattern, early warning signs, and why early referral matters. Screening Newborn screening What screening can and cannot tell you, and what follow-up tests are used. Treatment Current treatments overview HSCT, ERT, and supportive care explained in one place. Supportive care Supportive care and specialists What long-term multidisciplinary care often includes for families. Community Support and community Family support, connection, and practical resources.

Dawn Therapeutics status

Dawn Therapeutics is a preclinical biotechnology company working on tissue-targeted delivery platforms and a lead programme in Hurler syndrome. We do not have a clinical trial open at this time, but we are committed to sharing clear educational information and supporting respectful communication with families.

For medical decisions, diagnosis, treatment timing, or emergency care, always follow your metabolic specialist team, transplant team, and local treating clinicians.

Partner, clinician, or charity contact

If you are a clinician, patient charity, or support organisation and would like to collaborate on family education materials, you can use the same request form above and select your role in the form.

Contact Dawn Research hub

Educational information only. This page is intended to support patients and families with reliable information and signposting. It does not provide medical advice or replace your clinical team.