Rare diseases Clinical Trials: Active Studies by Market and Country

• 11markets with active trials
• 356active studies on Amyloidosis Treatment
• 30of our places with active trials

Source: ClinicalTrials.gov, Studies recruiting, active or not yet recruiting, worldwide. Retrieved 12 Nov 2026.

1. 1Amyloidosis Treatment356Amyloidosis Treatment: See them on ClinicalTrials.gov (opens in a new tab)
2. 2ALS Treatment279ALS Treatment: See them on ClinicalTrials.gov (opens in a new tab)
3. 3Sickle Cell Disease Therapeutics268Sickle Cell Disease Therapeutics: See them on ClinicalTrials.gov (opens in a new tab)
4. 4Cystic Fibrosis Therapeutics237Cystic Fibrosis Therapeutics: See them on ClinicalTrials.gov (opens in a new tab)
5. 5Hemophilia Treatment170Hemophilia Treatment: See them on ClinicalTrials.gov (opens in a new tab)
6. 6Myasthenia Gravis Treatment150Myasthenia Gravis Treatment: See them on ClinicalTrials.gov (opens in a new tab)
7. 7Spinal Muscular Atrophy Treatment131Spinal Muscular Atrophy Treatment: See them on ClinicalTrials.gov (opens in a new tab)
8. 8Duchenne Muscular Dystrophy Treatment116Duchenne Muscular Dystrophy Treatment: See them on ClinicalTrials.gov (opens in a new tab)
9. 9Huntington's Disease Treatment66Huntington's Disease Treatment: See them on ClinicalTrials.gov (opens in a new tab)
10. 10Phenylketonuria Treatment43Phenylketonuria Treatment: See them on ClinicalTrials.gov (opens in a new tab)
11. 11Gene Therapy for Hemophilia24Gene Therapy for Hemophilia: See them on ClinicalTrials.gov (opens in a new tab)

Source: ClinicalTrials.gov, Studies recruiting, active or not yet recruiting, worldwide. Retrieved 12 Nov 2026.

• A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis

  NCT07298044Amyloidosis Treatment

• VR Pupillometry in Cognitive Impairment

  NCT07402356Amyloidosis Treatment

• Elevated Rate Pacing of Cardiac Amyloidosis

  NCT07583823Amyloidosis Treatment

• Imaging Biomarkers in ALS

  NCT02567136ALS Treatment

• REstoration of Communication and LAnguage With Intracortical Microelectrodes

  NCT07933549ALS Treatment

• Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

  NCT03225144ALS Treatment

• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

  NCT04610866Sickle Cell Disease Therapeutics

• mAnaging siCkle CELl disEase Through incReased AdopTion of hydroxyurEa in Nigeria

  NCT06318143Sickle Cell Disease Therapeutics

• Natural History of Sickle Cell Disease

  NCT00081523Sickle Cell Disease Therapeutics

• Continuous Glucose Monitoring Parameter and Oral Glucose Tolerance Testing (OGTT) Parameters in Children and Young Adults With Cystic Fibrosis (CF)

  NCT07930884Cystic Fibrosis Therapeutics

• The Effects of Yoga Practice on Exercise Capacity, Arterial Stiffness, Respiratory Function, Peripheral Muscle Strength, Posture and Quality of Life in Children With Cystic Fibrosis

  NCT07930221Cystic Fibrosis Therapeutics

• Natural History of Bronchiectasis

  NCT00943514Cystic Fibrosis Therapeutics

• The CACHE Study: Coronary Artery Care in HaEmophilia

  NCT06706206Hemophilia Treatment

• A Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, PK/PD Characteristics, and Efficacy of SCTB10

  NCT07847697Hemophilia Treatment

• A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

  NCT05568459Hemophilia Treatment

• An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis

  NCT06540144Myasthenia Gravis Treatment

• A Study to Evaluate The Long-Term Safety And Efficacy of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

  NCT07465289Myasthenia Gravis Treatment

• A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

  NCT07463521Myasthenia Gravis Treatment

• A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

  NCT05789758Spinal Muscular Atrophy Treatment

• A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

  NCT06019637Spinal Muscular Atrophy Treatment

• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia

  NCT07737756Spinal Muscular Atrophy Treatment

• Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy

  NCT07642635Duchenne Muscular Dystrophy Treatment

• Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

  NCT07542314Duchenne Muscular Dystrophy Treatment

• Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim

  NCT06093100Duchenne Muscular Dystrophy Treatment

Source: ClinicalTrials.gov, Study records, newest update first. Retrieved 12 Nov 2026.

• Genomics
• Brain and nerves
• Biologics and biosimilars
• Diagnostics and lab testing
• Clinical trials
• Cardiovascular
• Respiratory
• Pediatric
• Cell and gene therapy
• Autoimmune diseases