Rare diseases
Investigational molecules
Locations
50 Northern Avenue, 02210-1862, United States
Vertex Pharmaceuticals Inc. maintains a major clinical focus on cystic fibrosis, with studies spanning long-term treatment evaluation and combination regimens built around CFTR modulator therapies. Research activity includes interest in both established and newer treatment approaches across a broad age range, including infants, children, adolescents, and adults.
Its cystic fibrosis portfolio reflects sustained attention to respiratory disease management and disease-modifying therapy development.
Vertex Pharmaceuticals Inc. runs clinical trials in conditions such as Cystic fibrosis, Sickle cell anaemia, Myotonic dystrophy, Thalassaemia, among others.
Clinical trials run by this sponsor – those enrolling participants first.
Phase 2 Randomized Study of Povetacicept to Assess Safety and Effectiveness in Adults with Generalized Myasthenia Gravis
PolandA Study of Povetacicept Compared to Tacrolimus for Adults with Primary Membranous Nephropathy
CzechiaStudy on Long-Term Safety and Efficacy of VX-670 for Adults with Myotonic Dystrophy Type I
BelgiumStudy on the Effectiveness and Safety of VX-993 and Pregabalin for Pain Relief in Patients with Diabetic Peripheral Neuropathy
FranceStudy on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor
GermanyStudy on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination
GermanyStudy of VX-121, Tezacaftor, and Deutivacaftor for Children Aged 1-11 with Cystic Fibrosis
BelgiumStudy on the Safety and Effectiveness of VX-880 for Patients with Type 1 Diabetes and Severe Low Blood Sugar Awareness
FranceStudy on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1
BelgiumLong-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
BelgiumStudy on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
GermanyStudy on Long-Term Safety and Effectiveness of Vanzacaftor, Tezacaftor, and Deutivacaftor for Cystic Fibrosis in Patients Aged 1 Year and Older
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