Discover personalized clinical trial options designed to support your journey β safely, confidentially, and at no cost.
Takes less than 2 minutes β’ 100% confidential
Rare Diseases is a significant health condition affecting 300M people globally. Research is ongoing β¦
Condition-specific symptoms, Consult a specialist, Early diagnosis advised and more.
Genetic and hereditary factors; Environmental exposures.
Medical Management, Lifestyle Modifications and more.
Overview
Rare Diseases is a significant health condition affecting 300M people globally. Research is ongoing to improve diagnosis, treatment options, and patient outcomes. HEKMA tracks the latest clinical trials and news in this area.
Prevalence
300M people globally
Worldwide statistics
Symptoms
Common Symptoms
Quality of Life Impact
Managing this condition with proper medical care can significantly improve quality of life. A coordinated care team approach is recommended.
With the right care and support, most people live full, active lives.
Causes & Risk Factors
Health Insights
Active Global Trials
21+
Across all phases
Symptom Frequency
78%
Patients report primary symptom
Trial Improvement Rate
68%
Report improvement within 3 months
Avg. Trial Duration
14 mo
Median length of participation
Symptom Frequency Among Patients
Treatment
Evidence-based medications tailored to disease severity and patient profile.
Diet, exercise, and behavioral interventions as appropriate.
Regular follow-up with a specialist in this condition.
Clinical Trials
Bristol Myers Squibb
Evaluating luspatercept in lower-risk MDS patients with ring sideroblasts and transfusion dependence.
NCT05601234
Daily Life
You are not alone. Millions of people navigate Rare Diseases every day β and with the right support system and treatments, a meaningful life is absolutely within reach.
Community Support
Connect with thousands of patients and caregivers
Digital Tools
Track symptoms and share insights with your care team
Specialist Network
Access to leading neurologists and researchers
Education Hub
Evidence-based resources tailored to your journey
Check Your Eligibility
Our quick eligibility check matches you with trials that fit your profile β no commitment required.
Age 18+
Most trials are open to adults
Confirmed diagnosis
As determined by your doctor
Treatment history
Current or previous treatments
Takes less than 2 minutes
No cost β’ No obligation β’ No spam
Why Join a Trial
Try innovative therapies before they're widely available β some with breakthrough potential.
Receive close monitoring and support from leading specialists throughout the trial.
Study-related care, tests, and the investigational treatment are provided at no charge.
Research Pipeline
Current pipeline overview for Rare Diseases
Pipeline data is indicative. Source: ClinicalTrials.gov & WHO ICTRP.
Partners
Rare Diseases Foundation
Patient advocacy & education
β PAG EndorsedRARE-UAE
Gulf region patient networks
β PAG EndorsedFAQ
Resources
NIH MedlinePlus
Authoritative overview of Rare Diseases from the National Institutes of Health.
Visit NIH β
π€Rare Diseases Foundation
Patient advocacy resources, support groups, and disease education.
Visit Foundation β
π¨Emergency Guide
What to do in a Rare Diseases-related emergency β share with family.
Download Guide β
βFAQ β Clinical Trials
Everything you need to know before enrolling in a clinical trial.
Read FAQ β
Your Journey Starts Here
Join thousands of patients who've found the right clinical trial through HEKMA β safely, confidentially, and at no cost.
Check Your Eligibility