TürkiyeResearch and clinical trials
Gene therapy given to a patient with severe hemophilia B at Gaziantep University
A patient diagnosed with severe hemophilia B received gene therapy at Gaziantep University Hospital.
Blood and immune system diseases
An inherited bleeding disorder. A factor that helps the blood to clot is missing. If factor VIII is missing, it is hemophilia A; if factor IX is missing, it is hemophilia B.
Not part of the panel of Türkiye's National Newborn Screening Programme (heel-prick test). Source
Orphanet has a guideline for emergency doctors for this disease (language: German, French, Italian). See on Orphanet
This page is translated from the Turkish original.
In hemophilia, a protein that makes blood clot is missing or does not work well. This protein is called a clotting factor. If factor VIII is missing, it is hemophilia A; if factor IX is missing, it is hemophilia B. Because of this, bleeding lasts a long time and is hard to stop.
Symptoms often begin in early childhood. In the severe type, bleeding can also happen on its own. The mild type may not be noticed until an operation or a serious injury.
Symptoms, or hemophilia in the family, raise suspicion. Blood tests check how long clotting takes. Factor VIII and IX levels are measured. This level shows how severe the disease is.
The main approach is to replace the missing clotting factor. This treatment can be given regularly to prevent bleeding, or when bleeding happens. For hemophilia A, there are also non-factor treatments. New approaches such as gene therapy are also being developed. The doctor who follows the disease decides which treatment is suitable.
Hemophilia is passed on through the X chromosome and mostly affects males. For each son of a carrier mother, the chance of having the disease is 50%. For each daughter, the chance of being a carrier is also 50%. Women who are carriers may have mild symptoms. Genetic counselling guides the family for future pregnancies.
Source: MedlinePlus Genetics — Hemophilia · Orphanet — Hemophilia (ORPHA:448) · NHS — Haemophilia
These facts are taken from Orphanet and MedlinePlus records (last checked: October 7, 2026). Frequency values are approximate ranges.
Non-profit patient and family organizations in Türkiye working on this disease or disease group. Links are for information; Doktorclub is not affiliated with these organizations and does not endorse them.
For all rare diseases: Nadir Hastalıklar Federasyonu
Links checked on October 8, 2026.
Doktorclub does not diagnose, recommend treatments or refer you to hospitals or doctors. These pages are meant to help you talk with your doctor.
TürkiyeResearch and clinical trials
A patient diagnosed with severe hemophilia B received gene therapy at Gaziantep University Hospital.
EUNew treatments and approvals
The European Commission extended the use of marstacimab to also cover patients with hemophilia A with factor VIII inhibitors and hemophilia B with factor IX inhibitors.
InternationalResearch and clinical trials
Results of a phase 3 study of denecimig, given under the skin, in hemophilia A have been published.
USANew treatments and approvals
The FDA approved concizumab, previously approved for patients with inhibitors, also for preventing bleeding in patients aged 12 years and older with hemophilia A and B without inhibitors.
USANew treatments and approvals
The FDA has approved fitusiran to prevent bleeding in patients with hemophilia A or B.
TürkiyeResearch and clinical trials
According to the Federation of Hemophilia Associations, 46 hemophilia patients in Türkiye received gene therapy in clinical trials.
A clinical trial is a regulated study that tests whether a new treatment is safe and whether it works, in patients who volunteer. Most trials look for a certain age, genetic change and health condition. Taking part is voluntary.
Questions you can ask your doctor
ClinicalTrials.gov lists 97 active interventional studies for this disease. 44 are recruiting now; 17 have sites in Türkiye. Last checked: October 7, 2026.
Selected records (studies with a site in Türkiye first):
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT05568719
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT05611801
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT06003387
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT07285460
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT05145127
See all studies on ClinicalTrials.gov · Joining clinical trials
GlobalHealth policy
The International Pediatric Nephrology Association published its recommendations for diagnosing and treating atypical HUS in children.
GlobalResearch and clinical trials
Researchers re-examined the data from a large drug study.
TürkiyeResearch and clinical trials
A face-to-face survey was conducted with 104 PNH patients at 18 centers in Türkiye.
These codes identify the disease in international classifications and databases. They can help when you talk with your doctor or look for reliable sources.
Orphanet code: ORPHA:98878, ORPHA:98879 · ICD-11 3B10.0, 3B11.0
Codes checked against WHO ICD-11 and Orphanet on October 6, 2026.
This page provides general information and does not replace diagnosis or treatment. Talk to your doctor about symptoms, diagnosis and treatment.