PTC Therapeutics — Biomedical Watch V2

Traitements4programmes

Essais2liés

Publications12liées

SourceDBlocale

Traitements

4

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
AtalurenDuchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.	Myopathies	Phase 2	À vérifier	United States, United Kingdom	À vérifier
Chronic Corticosteroid TherapyDuchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.	Myopathies	Phase 2	À vérifier	United States, United Kingdom	À vérifier
AtalurenDuchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.	Myopathies	Phase 2	À vérifier	United States, United Kingdom	À vérifier
AtalurenDuchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.	Myopathies	Phase 2	À vérifier	United States	À vérifier

Essais cliniques

2

Molécule	Indication / population	Phase	NCT	Titre	Statut
Ataluren	Myopathies	Phase 2	NCT04336826	A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	COMPLETED
Ataluren	Myopathies	Phase 2	NCT01009294	Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)	TERMINATED

Publications

12

Molécule	Indication / population	Titre	Journal	Date
Ataluren		Identification of novel small molecule compounds with readthrough activity in Nagashima-type palmoplantar keratosis.	Journal of dermatological science
Chronic Corticosteroid Therapy		Fat Embolism Syndrome Following Elective Orthopedic Surgery in a Patient With Duchenne Muscular Dystrophy.	Cureus
Ataluren		Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population.	Genes
Chronic Corticosteroid Therapy		Lycopene attenuates dexamethasone induced depression like behavior and immunological dysfunction via restoration of neuro-immune-metabolic homeostasis in rats.	Scientific reports
Ataluren		Patient-derived cornea organoids as drug repurposing models for aniridia-associated keratopathy.	Life sciences
Chronic Corticosteroid Therapy		Adrenal rather than central dysfunction limits HPA axis recovery after chronic glucocorticoid treatment in male mice.	Endocrinology
Chronic Corticosteroid Therapy		Community-acquired pneumonia outside the intensive care unit: Clinical characteristics and impact of rapid molecular diagnostics in the Italian SIS-NET study.	International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases
Chronic Corticosteroid Therapy		Occult Necrotizing Fasciitis Presenting as a Severe Hyperosmolar Hyperglycemic State in a Patient With Newly Diagnosed Type 2 Diabetes Mellitus.	Cureus
Ataluren		Ion-Channel-Mediated Drug Repurposing Opportunities Validated by Single-Cell Perturbation in Colorectal Cancer.	International journal of molecular sciences
Ataluren		CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).	The Cochrane database of systematic reviews
Chronic Corticosteroid Therapy		Very late stent thrombosis associated with stent malapposition and uncovered struts after contrast-enhanced CT in a patient receiving long-term steroid therapy due to Kounis syndrome: a case report.	European heart journal. Case reports
Chronic Corticosteroid Therapy		New Cortisol Thresholds for the Diagnosis of Adrenal Insufficiency Using the Low-Dose Synacthen Test in Children on Long-Term Corticosteroids: A North African Pilot Study.	Diagnostics (Basel, Switzerland)