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
Rare Diseases
News
New treatments, research and health policy from around the world and from Türkiye. Each with its source and level of evidence.
15 updates
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
TürkiyeSGK and reimbursement
Reimbursement criteria for nusinersen and risdiplam in SMA revised
The criteria for the use and reimbursement of nusinersen and risdiplam in SMA treatment have been revised.
Official statement or regulationSMA
EUHealth policy
European rare disease conference in Prague calls for a joint EU action plan
The 13th European Conference on Rare Diseases and Orphan Products (ECRD), held in Prague on June 3–4, 2026, came together in calling for a coordinated EU-wide action plan.
Official statement or regulation
GlobalHealth policy
Rare Disease Day 2026: 'More than you can imagine'
Rare Disease Day on February 28, 2026, was marked under the theme 'More than you can imagine', coordinated by EURORDIS.
Official statement or regulation
USAHealth policy
FDA leaders propose a 'plausible mechanism' pathway for individualized therapies
Writing in NEJM, FDA leaders described an approval pathway called the 'plausible mechanism' pathway for individualized therapies for which randomized trials cannot be conducted.
Official statement or regulation
United KingdomDiagnosis and screening
25,000 babies join England's newborn genome screening study
25,000 babies have joined the Generation Study, run by Genomics England with the NHS.
Official statement or regulation
USAHealth policy
US FDA announces new evidence principles for very rare genetic diseases
The US Food and Drug Administration (FDA) announced 'Rare Disease Evidence Principles' for genetic diseases that generally have fewer than 1,000 patients in the USA.
Official statement or regulation
USAResearch and clinical trials
New center launched for individualized gene-editing therapies for children
The Chan Zuckerberg Initiative and the Innovative Genomics Institute announced that they have set up a new center.
Official statement or regulation
United KingdomHealth policy
England's 10-year health plan aims to offer genome testing to newborns
The English government's 10-year health plan aims, in the long term, to offer genome sequencing at birth to everyone.
Official statement or regulation
TürkiyeSGK and reimbursement
SGK to add a drug used in cystic fibrosis treatment to reimbursement
SGK (Social Security Institution) announced that it will add a drug used to treat cystic fibrosis to the reimbursement list.
Official statement or regulationCystic fibrosis
GlobalHealth policy
World Health Assembly unanimously adopts its first resolution on rare diseases
The 78th World Health Assembly, led by Egypt and Spain, adopted the first resolution recognizing rare diseases as a global health priority.
Official statement or regulation
TürkiyeSGK and reimbursement
A third SMA drug to be added to the reimbursement list, minister announces
Another drug used to treat children with SMA will be added to the SGK (Social Security Institution) reimbursement list.
Official statement or regulationSMA
TürkiyeSGK and reimbursement
16 drugs added to SGK reimbursement list, including one for a genetic nerve disease
With an amendment to the Sağlık Uygulama Tebliği (SUT, Health Implementation Communiqué), 16 drugs were added to the SGK (Social Security Institution) reimbursement list.
Official statement or regulation
TürkiyeHealth policy
2025 Annual Program: more rare disease centers, neuromuscular units to reach 30
The Ministry of Health will increase the number of treatment and research centers for rare diseases.
Official statement or regulation