South KoreaResearch and clinical trials
86 LAM patients from South Korea: lung function is mostly preserved at diagnosis
In South Korea, 86 women with LAM from 20 centers were studied.
Peer-reviewed publicationLAM
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Latest news
312 updates · 140 from Türkiye · 172 worldwide. Sources checked on October 7, 2026.
TürkiyeResearch and clinical trials
Children with PCD at Hacettepe: lung function values lower in those with Pseudomonas in the lungs
Hacettepe University reviewed the records of 290 patients with PCD.
Peer-reviewed publicationPCD
InternationalResearch and clinical trials
Experimental oral RIPK1 inhibitor showed no benefit in ALS in a phase 2 trial
The HIMALAYA study, published in The Lancet Neurology, examined an experimental drug in patients with ALS (amyotrophic lateral sclerosis).
Peer-reviewed publicationALS
EUHealth policy
European Parliament health committee calls for a rare disease action plan
The European Parliament's Public Health Committee adopted, with 33 votes, a report calling on the European Commission to draw up an action plan for rare diseases.
Official statement or regulation
GlobalResearch and clinical trials
Garetosmab markedly reduced new bone formation in FOP in a phase 3 trial
The trial included 63 adults from 16 countries.
Peer-reviewed publicationFOP
InternationalResearch and clinical trials
In Hunter syndrome, siblings who started enzyme therapy in infancy did better
Records of 19 sibling pairs with Hunter syndrome were compared.
Peer-reviewed publicationMPS II
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From Türkiye
SGK (Social Security Institution) decisions, screening programmes and research in Türkiye.
TürkiyeResearch and clinical trials
Children with PCD at Hacettepe: lung function values lower in those with Pseudomonas in the lungs
Hacettepe University reviewed the records of 290 patients with PCD.
Peer-reviewed publicationPCD
TürkiyeSGK and reimbursement
SGK adds a drug for a rare disease affecting the optic nerve to reimbursement
A drug used for a rare disease affecting the optic nerve has been added to reimbursement coverage.
Official statement or regulation
TürkiyeResearch and clinical trials
Guillain-Barré after COVID-19 in Istanbul: the type affecting the nerve sheath was more common
A multicenter study in Istanbul compared patients with Guillain-Barré syndrome.
Peer-reviewed publicationGuillain-Barré
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Not all information carries the same weight
That is why we label every update by its level of evidence. From strongest to weakest:
- Peer-reviewed publication
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
- Official drug approval
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
- Preliminary result
An early result that has not yet been published in a peer-reviewed journal, or comes from a small, uncontrolled study. Treat it with caution.
- Official statement or regulation
A statement by a ministry, institution or international organisation, or a change in regulations. It is not evidence about how well a treatment works.
- News report
Based on a single news source or an institutional announcement. The level of scientific evidence is low; check the details with a doctor.
You are not alone
at least 5 millionpeople in Türkiye estimated to be affected by a rare disease (AA, February 2026)
more than 300 millionpeople worldwide living with a rare disease (EURORDIS, 2026)
6diseases included in Türkiye’s newborn heel-prick screening (Turkish Ministry of Health)