Severe hemophilia A is a form of hemophilia A characterized by a large deficiency of factor VIII leading to frequent spontaneous hemorrhage and abnormal bleeding as a result of minor injuries, or foll...
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
The limited documentation surrounding severe hemophilia A is primarily due to its rarity, affecting fewer than 10 in 100,000 people. This low prevalence restricts the scope of systematic clinical studies and comprehensive phenotype documentation. Additionally, the genetic underpinnings of the condition remain largely unexplored, making it challenging to establish a detailed clinical profile.
To navigate your care effectively, consider consulting a hematologist with expertise in bleeding disorders, particularly hemophilia. Engaging with specialized treatment centers can provide access to cutting-edge therapies and clinical trials. For additional resources, the National Organization for Rare Disorders (NORD) offers valuable information at rarediseases.org. While no patient organizations are currently identified, participating in clinical trials may provide opportunities for support and access to new treatments.
There are currently 48 active clinical trials focusing on severe hemophilia A, exploring various treatment options and therapies. Notable orphan drugs in development include gene therapies and novel factor VIII replacements. These investigational treatments aim to improve patient outcomes significantly. For more information on ongoing trials, visit ClinicalTrials.gov: https://clinicaltrials.gov/search?cond=severe%20hemophilia%20A.
Actionable guidance for navigating care for severe hemophilia A
To navigate your care effectively, consider consulting a hematologist with expertise in bleeding disorders, particularly hemophilia. Engaging with specialized treatment centers can provide access to cutting-edge therapies and clinical trials. For additional resources, the National Organization for Rare Disorders (NORD) offers valuable information at rarediseases.org. While no patient organizations are currently identified, participating in clinical trials may provide opportunities for support and access to new treatments.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 3 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning severe hemophilia A
Updated Feb 6, 2026
Pfizer has initiated a Phase 3 trial for giroctocogene fitelparvovec (PF-07055480), a gene therapy targeting severe hemophilia A. This advancement reflects Pfizer's commitment to developing transformative treatments for rare diseases.