The daily injectable vosoritide appears promising for various genetic causes of short stature in children, new phase 2 trial data suggested.
"We've had patients travel from literally all over the world to be a part of our trials because they're so motivated and excited by this opportunity to have a therapy which can help their children grow. That's been really gratifying and exciting to be a part of," Dauber said. Wasserman pointed out,"In any kind of condition where growth is being treated, you might see an initial gain in that first year. Even with growth hormone, you get your best response in that first year, and then, it tapers off. And then of course, ultimately, the question is going to be how does this impact final adult height. Just because you increase their growth velocity doesn't mean that it has a sustained effect on final adult height.
In addition, scoliosis was reported in seven patients, of which four cases were present prior to treatment.
GH Deficiency Growth Hormone Deficiency (GHD) GHD - Growth Hormone Deficiency Children Child Childhood Pediatrics Kids Genomics Genomic Medicine Genetics Short Stature Growth Hormone Genetic Testing Scoliosis Washington DC Washington - District Of Columbia Achondroplasia Hospitals Washington Endocrinology Endocrine System Biologic Therapy Biologics Bones
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