THE FAMILIES OF CHILDREN WITH SMA ARE IN A DIFFICULT SITUATION...

Ayhan KIZILTAN, ben@ayhankiziltan.com, Mersin, Türkiye, 26.09.2025

Yesterday, I was visited by the grandfather, father, uncle, and a close friend of mine, relatives of baby Alim Asaf Konat, who suffers from a rare disease with extremely costly treatment: SMA.

They explained Alim Asaf 's situation. Through their efforts, they have managed to collect 38% of the estimated 2 million dollars required for treatment. We must thank the compassionate and benevolent people who have supported them.

They were in a state of despair, anxiety, and sorrowful haste; especially the grandfather…“I was once wealthy, but after facing many difficulties, I lost everything. I want to remain hopeful to keep my grandson alive,” he said, with tears in his eyes.

The baby’s father, Latif Konat, described how desperately he has been trying to make his voice heard and secure support for his child. He said it is very difficult to reach influential and authoritative people. “In order to reach them, I hold up my child’s poster at events and raise my voice in public. We are not beggars; we only seek to save our child’s life.”

The Metropolitan Mayor has significantly supported Alim Asaf through a live TV campaign, by the effort of the father of Alim Asaf.

Like every child, Alim Asaf is very charming. He was born in Mersin, Türkiye on February 15, 2021. At 27 months old, he was diagnosed with SMA.

Every newborn is the child of the state until reaching a certain age. The state is obliged to care for them. With this understanding, the treatment and medication expenses of Rare Diseases like SMA must be covered by the state.

No family should have to beg for help on the streets, knock on businesspeople’s doors, or chase after politicians for their child’s right to live.

IF YOU WOULD LIKE TO DONATE TO “ALIM ASAF KONAT”, THE INFORMATION IS BELOW:

STATE-AUTHORIZED DONATION BANK ACCOUNTS:

Recipient : ALIM ASAF KONAT

TL          : TR10 0006 4000 0016 6050 5466 62

USD       : TR14 0006 4000 0029 6050 1465 57

EUR       : TR31 0006 4000 0026 6050 1465 42

E-GOVERNMENT VERIFICATION INFO:

Activity No      : 33.2024.2421

Name             : ŞÜKRAN KONAT

Current Situation of SMA and Rare Diseases in Turkey

• There are approximately 3,000 SMA patients in Turkey. Every year, about 150 babies are born with SMA.

• Treatments:

- Spinraza (Nusinersen): Covered by Turkish Social Security, free of charge   

- Risdiplam (Evrysdi): Covered by Turkish Social Security as of 2025   

- Zolgensma (Gene Therapy): World’s most expensive treatment (~2 million USD), not yet covered by Social Security in Turkey

• Families are forced to organize fundraising campaigns for Zolgensma.

Today, there is still no definitive cure for SMA. However, SMA is no longer an inevitably fatal disease. With early diagnosis + appropriate drug therapy + intensive physiotherapy, many children and young people can sustain independent lives.

The most effective treatment is the Zolgensma cure, which costs around 2 million USD.

Existing State Support

The Turkish Government provides many supports for Rare Diseases:

• Spinraza and Risdiplam treatments are free. 

• Medical devices such as ventilators and wheelchairs are supplied by Turkish Social Security. 

• Home care allowance, disability pensions, and tax exemptions are applied. 

• SMA carrier tests before marriage are provided free of charge.

In addition to state support, NGOs also provide supplementary assistance:

• Medical devices & supplies (ventilators, equipment, consumables) via foundations/associations 

• Rent support for some families 

• Education scholarships for SMA patients and their relatives 

• Stationery and other social support programs

Problems

• No access to Zolgensma 

• Families are forced to hold fundraising campaigns 

• Income differences create inequality 

• Similar problems exist for other rare diseases

Campaigns also face debates:

• Public support: Many segments of society, including celebrities and social media influencers, participate. 

• Transparency issues: In some campaigns, fund collection and usage became controversial. Hence, campaigns are subject to official permission and monitoring. 

• Psychological effects: Families face emotional burden by having to constantly appeal for help on social media.

International Examples

• France: National Rare Disease Plans, reference centers, state funding 

• Germany: NAMSE network, treatments under insurance coverage 

• UK: NICE-HST program, Innovative Medicines Fund 

• Common point: Rare Disease Funds and solidarity-based approachZolgensma is applied in the USA, Europe, Japan, and many developed countries. 

In Turkey, it is not yet licensed, therefore not covered by Turkish Security or routinely applied.  It can only be applied in limited cases with special approval from the Ministry of Health.

Proposed Model for Turkey: Rare Disease Fund

• Purpose: To ensure high-cost rare disease treatments are guaranteed by the state 

• Financing: General health insurance + Treasury contribution + small co-payment option; risk-sharing with producers 

• Governance: Special Rare Diseases Department under Turkish Social Security 

• Early Diagnosis: Genetic tests can detect SMA during pregnancy 

• Infrastructure: National patient registry, newborn screening, application centers 

• Performance: Access time, clinical outcomes, geographic equity, transparent reporting

Financial Simulation (Summary)

There are about 26 million Turkish Social Security -insured people in Turkey.

• If 3,000 SMA patients receive Zolgensma: additional monthly premium per person ≈ 90–120 TL 

• If only 150 newborns per year receive treatment: additional monthly premium ≈ 30–40 TL per person 

• This creates a sustainable fund at a reasonable cost 

• These premiums must be secured under a “Rare Disease Treatment Fund” protected by the Constitution, and cannot be used elsewhere

Conclusion and Call

Turkey must establish a Rare Disease Fund based on the principle that every child is the child of the state.  No family should be forced to organize street fundraising campaigns.  All children should have equal and fair access to healthcare.  This fund will be one of the strongest indicators of a social state.

In cities, individual campaigns rarely succeed without the support of influential figures (governors, mayors, chamber presidents, prominent individuals…).

Live TV campaigns, social media posts, and donation stands in busy streets unfortunately do not solve the suffering of these children.

Until the state takes serious structural action, in the short term, “Rare Disease Support Pools” should be established under the leadership of governors and mayors. Just as businesspeople and corporations are called upon to support sports clubs, they should also be called upon to support the Rare Disease Pool.

Every child struggling with SMA or any other rare disease is a child of society, and therefore of the state.

THE STATE MUST ENSURE CHILDREN’S RIGHT TO LIFE.