How to raise money for rare disease treatment

Living with a rare disease can mean years of rehabilitation, specialised therapies and everyday care. For many patients and their families, these needs also come with significant costs. Fundraising can help cover them. In this guide, you’ll find examples of rare diseases and fundraisers created to support people living with them. You’ll also learn what you can raise money for and how to start a fundraiser for yourself or someone close to you.

Rare disease treatment – Key Facts

• In the European Union, a disease is considered rare when it affects fewer than 5 in 10,000 people.
• There are an estimated 8,000 different rare diseases.
• Around 36 million people in the EU live with a rare disease.
• Around 95% of known rare diseases still have no approved treatment.
• Some rare disease treatments can cost over €1 million per year – on 4fund.com people can raise funds for treatment without mandatory fundraising fees.

What is a rare disease

A rare disease is a condition that affects fewer than 5 in 10,000 people in the European Union. Although each individual disease affects a relatively small number of people, rare diseases collectively affect millions of Europeans and include genetic, neurological, autoimmune, metabolic and oncological conditions.

For people living with a rare disease, access to diagnosis, specialist care and treatment can be challenging. Medical care, rehabilitation, equipment and travel to specialist centres can also create significant costs – which is why some patients and their families turn to fundraising for additional support.

Below, we present a list of rare genetic diseases that can be supported through fundraising.

Rare genetic diseases

Around 80% of rare diseases have a genetic origin. They can affect different organs and systems, and their symptoms and severity may vary widely from one person to another.

Usher syndrome

Usher syndrome is a rare inherited condition that causes hearing loss and progressive vision loss. Some types also affect balance, which can make learning to sit or walk more difficult.

Inspired by Emma’s diagnosis, her godfather William and Lara launched a fundraiser to support research into Usher syndrome type 1B and the development of future treatments. The campaign has already reached its fundraising goal, raising over €10,000.

Andersen disease

Andersen disease, also known as glycogen storage disease type IV (GSD IV), is a rare inherited metabolic disorder in which abnormally formed glycogen builds up in cells. It can damage the liver and muscles, although symptoms and severity vary between its different forms.

Julia’s mother, Szilvia, set up a fundraiser for a procedure in Barcelona intended to address her daughter’s joint contractures, which restrict her movement.

Prader–Willi syndrome (PWS)

Prader-Willi syndrome (PWS) is a rare genetic condition that affects development, muscle tone, growth and appetite. Babies often experience feeding difficulties, while persistent hunger typically develops during childhood and can make food intake difficult to manage.

Vedran and his family have already raised over €20,000 through their fundraiser for their daughter, who has both PWS and calpainopathy.

Angelman syndrome

Angelman syndrome is a rare genetic condition that affects the nervous system, causing developmental delays, significant speech difficulties and problems with movement and balance. Many people with the condition also experience seizures and sleep difficulties.

Florijan loves music, books and spending time with others. His fundraiser helps cover regular therapies that his family largely pays for privately, supporting his development and everyday functioning.

Spinal muscular atrophy (SMA)

Spinal muscular atrophy (SMA) is a rare genetic condition in which the loss of nerve cells controlling movement leads to muscle weakness and wasting. Depending on its type and severity, it can also affect swallowing and breathing.

Ștefan, who has SMA type 1, received gene therapy as a baby but still needs ongoing rehabilitation. His family is raising money for regular physiotherapy and specialist rehabilitation sessions.

YWHAG

YWHAG is an extremely rare genetic disorder that affects the development and functioning of the nervous system. It can cause, among other symptoms, epilepsy, developmental delays, and difficulties with speech and independent movement. According to the YWHAG Research Foundation, 63 confirmed cases of people with a YWHAG gene mutation have been officially registered worldwide, including 25 in Europe.

Today, Rafael is the only child in Greece diagnosed with YWHAG.

“The road to diagnosis was very long. In Greece, none of the doctors we had previously consulted were really familiar with YWHAG. Even today, the condition remains extremely little known in our country,” says Rafael’s mother.

His mother started a fundraiser to support both research into YWHAG and her son’s everyday needs related to the condition.

Rare autoimmune diseases

Rare autoimmune diseases occur when the immune system mistakenly attacks the body’s own tissues. Depending on the condition, they can affect the nervous system, blood vessels, lungs, kidneys or other organs.

Granulomatosis with polyangiitis (GPA)

Granulomatosis with polyangiitis (GPA), formerly known as Wegener’s granulomatosis, is a rare autoimmune disease that causes inflammation of the blood vessels. It can affect several organs, particularly the respiratory tract and kidneys.

Anastasia was diagnosed with GPA as a teenager. Her fundraiser was created for granulomatosis with polyangiitis treatment and everyday needs during her illness.

Guillain–Barré syndrome (GBS)

Guillain–Barré syndrome (GBS) is a rare autoimmune neurological disorder in which the immune system attacks the peripheral nerves. It can cause rapidly progressing muscle weakness and, in severe cases, paralysis and problems with breathing.

GBS left Fabio unable to move, speak or breathe independently. His fundraiser has already raised over €18,000, exceeding its original €14,000 goal. The funds support his long recovery and everyday living costs while he is unable to work.

Rare neurological diseases

Rare neurological diseases affect the brain, spinal cord, nerves or muscles and can lead to problems with movement, communication, development or other everyday functions.

Amyotrophic lateral sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurological disease that damages the nerve cells responsible for voluntary muscle movement. Over time, it can affect walking, speaking, swallowing and breathing.

Kasia’s fundraiser was created to help finance a stair lift, home adaptations, eye-tracking communication equipment, ongoing healthcare, physiotherapy and rehabilitation.

West syndrome

West syndrome is a rare epilepsy syndrome that begins in infancy and is characterised by epileptic spasms, abnormal EEG activity and developmental slowing or regression. It can have different underlying causes, including structural brain abnormalities and genetic conditions.

Emili lives with West syndrome as well as cerebral palsy following a prenatal stroke. Her fundraiser was created to help cover a planned treatment programme and intensive rehabilitation.

Neonatal hypoxic-ischemic encephalopathy (HIE)

Neonatal hypoxic-ischemic encephalopathy (HIE) is a serious condition caused by reduced oxygen and blood flow to a baby’s brain around the time of birth. It can result in brain injury and long-term developmental difficulties.

Gloria was diagnosed with HIE and severe hypotonia after birth. Her family’s fundraiser helps cover treatment, therapies and ongoing specialised care.

Rare cancers

Rare cancers are malignant diseases diagnosed in fewer than 6 people per 100,000 each year. Together, they account for around 20-25% of all new cancer diagnoses.

Clear cell sarcoma

Clear cell sarcoma is a rare type of soft tissue cancer. It most often develops in the arms or legs, but it can also occur in other parts of the body, including the gastrointestinal tract.

After clear cell sarcoma spread to his liver, Dimitris started a fundraiser to help cover urgent specialist surgery in Athens.

Acute myeloid leukemia (AML)

Acute myeloid leukemia (AML) is a rare cancer of the blood and bone marrow in which abnormal myeloid cells multiply rapidly and interfere with normal blood-cell production.

Ágoston was diagnosed with AML at just 3.5 months old. His family created a fundraiser to help cover the cost of hospital treatment in the United States.

How to get funding for rare disease treatment

The conditions and stories above are only a few examples of the thousands of rare diseases people live with. Other rare diseases include Duchenne muscular dystrophy (DMD), Rett syndrome, cauda equina syndrome (CES), cervico-thoracic neuroblastoma, systemic sclerosis, calpainopathy, idiopathic pulmonary fibrosis, myasthenia gravis and aplastic anemia.

You do not need to find your exact diagnosis on this list to start a fundraiser. You can raise money for needs such as:

• Treatment and diagnosis – medication, specialist consultations, tests and medical procedures,
• Rehabilitation and therapies – regular physiotherapy, communication therapy and other forms of support,
• Medical equipment and home adaptations – wheelchairs, communication devices and changes that make everyday life at home easier,
• Travel and accommodation – transport to specialist centres, accommodation and costs related to a caregiver’s stay,
• Care and everyday expenses – support at home and living costs during illness,
• Medical research – supporting organisations and research projects focused on a particular rare disease.

If you or someone close to you needs financial support, you can start a fundraiser and ask your community for help. Give others the opportunity to support you – or help someone you care about get the support they need.

Funding a rare disease treatment – FAQ

Find answers to common questions about raising funds for medical care and everyday support.

Can I raise funds for rare disease treatment on 4fund.com?

Yes. You can create a fundraiser for treatment, rehabilitation, medical equipment and other expenses related to a rare disease. There are no mandatory fees for creating or running a fundraiser, receiving donations or withdrawals.

Can I start a fundraiser for someone else with a rare disease?

Yes. You can organise a fundraiser to support a family member or someone close to you, provided you meet the platform's requirements and obtain any necessary consent from the beneficiary.

Can I raise funds for a rare disease that is not mentioned in this article?

Yes. The diseases described in this article are only examples. You can create a fundraiser for other rare conditions and explain what financial support is needed for treatment, rehabilitation or everyday care.

Is it safe to use 4fund.com?

Yes. At 4fund.com, we take steps to make fundraising safe and transparent for everyone. As a licensed payment services provider, we verify organisers' identities and review fundraisers related to medical treatment. To verify a fundraiser, we require supporting documents confirming its purpose. We may also check whether collected funds are used as intended. These measures help protect donors and build trust in fundraising.

What documents are needed to verify my fundraiser?

For medical fundraisers, we may request documents confirming the diagnosis, treatment needs or related expenses, such as medical records, treatment plans or invoices. The documents required depend on the fundraiser's purpose and verification needs.

This article is for informational purposes only and does not constitute medical advice. 4fund.com is a crowdfunding platform – we do not provide medical services or recommend specific treatments, clinics or therapies. Information about fundraisers comes from their organisers, who are responsible for its accuracy. Always consult a qualified healthcare professional about diagnosis and treatment.