Congenital, familial and genetic disorders – page 12

Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease

Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease

Study on the Safety of Intradermal mRNA COVID-19 Vaccine Bretovameran for Patients with Fibrodysplasia Ossificans Progressiva

Study on the Safety of Intradermal mRNA COVID-19 Vaccine Bretovameran for Patients with Fibrodysplasia Ossificans Progressiva

Study on the Effectiveness of Oxybutynin for Children with Overactive Neurogenic Bladder Due to Spina Bifida

Study on the Effectiveness of Oxybutynin for Children with Overactive Neurogenic Bladder Due to Spina Bifida

Study on the Effects of Full Spectrum Cannabis Extract (Dronabinol, Cannabidiol) for Patients with Hard-to-Treat Epilepsy in Tuberous Sclerosis Complex

Study on the Effects of Full Spectrum Cannabis Extract (Dronabinol, Cannabidiol) for Patients with Hard-to-Treat Epilepsy in Tuberous Sclerosis Complex

Study on the Effects of Sirolimus Cream for Patients with Port Wine Stains

Study on the Effects of Sirolimus Cream for Patients with Port Wine Stains

Study on How Nusinersen is Processed in Adults and Children with Spinal Muscular Atrophy Using the ThecaFlex DRx System

Study Comparing the Safety and Effectiveness of Mexiletine PR and Mexiletine IR for Adults with Non-Dystrophic Myotonias

Study Comparing the Safety and Effectiveness of Mexiletine PR and Mexiletine IR for Adults with Non-Dystrophic Myotonias

Study on Losartan and Hydrochlorothiazide for Osteogenesis Imperfecta in Patients Aged 16 and Above

Study on Losartan and Hydrochlorothiazide for Osteogenesis Imperfecta in Patients Aged 16 and Above

Study on the Safety and Effectiveness of Inclisiran in Children Aged 2 to Under 12 with Homozygous Familial Hypercholesterolemia

Study on the Safety and Effectiveness of Inclisiran in Children Aged 2 to Under 12 with Homozygous Familial Hypercholesterolemia

Study on the Effectiveness and Safety of Nizubaglustat in Patients with Niemann-Pick Type C, GM1, or GM2 Gangliosidosis

Study on the Safety and Effectiveness of Sirolimus and Vigabatrin for Preventing Symptoms in Infants with Tuberous Sclerosis Complex

Study on the Safety and Effectiveness of Sirolimus and Vigabatrin for Preventing Symptoms in Infants with Tuberous Sclerosis Complex

Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents