UCB Biosciences Inc.
- Address
- 4000 Paramount Parkway Suite 200
Sponsor Overview
Specializations
Rare Neuromuscular Disorders
Clinical research activity includes thymidine kinase 2 deficiency, with interest in therapies for inherited conditions that affect muscle function and cellular energy metabolism.
- Thymidine kinase 2 deficiency
- Inherited myopathy
- Mitochondrial disease
Work in this area centers on treatment options for patients with persistent neuromuscular impairment and related rare disease manifestations.
Developmental and Epileptic Encephalopathies
The sponsor supports studies in Dravet syndrome, reflecting a therapeutic focus on severe childhood-onset epileptic disorders with developmental consequences.
- Dravet syndrome
- Early-onset epilepsy
- Seizure control
Research interest extends to treatment approaches for infants and young children living with drug-resistant seizures and associated neurodevelopmental challenges.
Neurodevelopmental Disorders
Clinical trials also address Rett syndrome, with emphasis on conditions that combine neurological impairment, developmental regression, and complex functional needs.
- Rett syndrome
- Neurodevelopmental disability
- Motor and communication impairment
This area includes therapeutic interest in symptoms that affect movement, cognition, and day-to-day functioning in individuals with rare genetic neurodevelopmental disease.
Rare Disease Therapeutics
The sponsor’s portfolio spans orphan disease research with attention to treatments for genetically defined conditions affecting the nervous system and muscle.
- Orphan drug development
- Genetic disorders
- Pediatric rare disease
Its clinical research landscape reflects sustained involvement in specialty therapeutic areas where unmet medical need remains high.
Sponsored Trials
| Trial Name | Country | Status |
|---|---|---|
| Open-label, Single-arm, Phase 3 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to less Than 2 Years of Age with Dravet Syndrome |  Belgium Germany Italy Spain | Not Recruiting |
| Phase 2 Open-Label Study on Safety and Pharmacokinetics of Doxecitine and Doxribtimine in Thymidine Kinase 2 Deficiency Patients | Spain | Not Recruiting |
| Phase 3 Randomized, Double‑Blind, Placebo‑Controlled Study of Oral Fenfluramine Hydrochloride in Participants with Rett Syndrome | Belgium France Germany Hungary Italy Poland Spain | Recruiting |
Data & Insights
This sponsor works with 3 molecules, of which 0 are not being studied by other sponsors.
This sponsor has worked with 31 sites across their trial portfolio.
Therapeutic Focus
- 1.Congenital, Hereditary, and Neonatal Diseases and Abnormalities1 trial165 other sponsors
- 2.Musculoskeletal Diseases1 trial238 other sponsors
- 3.Nervous System Diseases1 trial449 other sponsors
Partnered Research Centres
31 centres- Address
- 264 Rue Saint Pierre, 13005 Marseille
- Address
- Jean Joseph Crocqlaan 15, 1020 Brussels
- Address
- Via Conte Ruggero 73, 94018 Troina
- Address
- Via Giovanni Battista Grassi 74, 20157 Milan
- Address
- Via Sergio Pansini 5, 80131 Naples
- Address
- Viale Gaetano Pieraccini 24, 50139 Florence
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