Congenital, familial and genetic disorders – page 31

Study of the effects of ibutamoren mesilate in prepubertal children with growth hormone deficiency

Study of the effects of ibutamoren mesilate in prepubertal children with growth hormone deficiency

Study of acetylsalicylic acid and diclofenac for treating blood clots in superficial venous malformations in children aged 6 to 17 years

Study of acetylsalicylic acid and diclofenac for treating blood clots in superficial venous malformations in children aged 6 to 17 years

Comparison of intrathecal baclofen pump and selective dorsal rhizotomy for children with cerebral palsy who cannot walk

Comparison of intrathecal baclofen pump and selective dorsal rhizotomy for children with cerebral palsy who cannot walk

Study of ASP2957 Gene Therapy for Male Patients with X-linked Myotubular Myopathy Who Require Breathing Support with Ventilators

Study of ASP2957 Gene Therapy for Male Patients with X-linked Myotubular Myopathy Who Require Breathing Support with Ventilators

Study of sodium butyrate enemas for bowel function recovery after surgery in children and adults with Hirschsprung's disease

Study of sodium butyrate enemas for bowel function recovery after surgery in children and adults with Hirschsprung's disease

A Study of Migalastat Safety and Effects in Children Aged 2 to 12 Years with Fabry Disease and Suitable Gene Changes

A Study of Migalastat Safety and Effects in Children Aged 2 to 12 Years with Fabry Disease and Suitable Gene Changes

Cannabidiol for behavioral problems in patients with Tuberous Sclerosis Complex, Sanfilippo syndrome, and Fragile X syndrome

Study of Zirconium-89 Girentuximab as a Diagnostic Tool for Patients with Von-Hippel Lindau Disease

Study of Zirconium-89 Girentuximab as a Diagnostic Tool for Patients with Von-Hippel Lindau Disease

Study of divarasib and pembrolizumab compared to standard chemotherapy in previously untreated KRAS G12C-mutated advanced non-small cell lung cancer

Study of divarasib and pembrolizumab compared to standard chemotherapy in previously untreated KRAS G12C-mutated advanced non-small cell lung cancer

First study of TTX-381 gene therapy safety in patients with eye problems caused by CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2)

First study of TTX-381 gene therapy safety in patients with eye problems caused by CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2)

Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders

Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders

Study on the Effects of Alglucosidase Alfa Enzyme Therapy in Children and Adults with Pompe Disease

Study on the Effects of Alglucosidase Alfa Enzyme Therapy in Children and Adults with Pompe Disease