Re: stifling disease in the womb: the first medical intrauterine injection was used to treat rare genetic diseases.
Corinna reached 21 weeks in the twin weeks of pregnancy and found that they had SLHED as well as their eldest son.
it is noteworthy that, although the drugs at the time of Edimer did not work for children, the clinical trial left a glimmer of hope for the treatment of pregnant animals to see the effect on their offspring.
Corinna and her husband then asked Schneider: is this intrauterine treatment feasible for its own twins?
"we were hesitant at that time. In that case, we must think twice. You will consider more risks. After all, there are 3 lives, but we will also take into account the hope of treatment. " Schneider says.
within a month after Corinna consultation, Schneider agreed to try treatment. El Langen Nuremberg University also approved treatment under the "sympathetic use". Schneider got the drugs left in the previous Edimer clinical trial.
it needs to be emphasized that the principle of intrauterine injection is that the protein needed to make the sweat glands is temporary and not needed, and the cycle is in 20 weeks to 30 weeks of pregnancy, which is the period of the formation of the sweat glands. Schneider said the treatment team injected the twins' amniotic sac directly.
Corinna believes that the treatment she received during pregnancy is effective. "Very successful, the twins can sweat normally." Although their facial features are somewhat abnormal, they also lack some teeth.
Anna David, director of the women's Health Institute at the University College London, UK, said, "the great point of this treatment is the critical time point for the development of the sweat glands in the uterus. I think this is the first time to see a protein drug correcting the genetic disease before the baby is born."
that is why other genetic diseases, such as hemophilia, will not be treated in the same way, because the body's needs for the missing molecules in hemophilia are permanent. Scientists may eventually consider using embryo gene therapy to deal with these diseases, but they have not yet tried.
of course, more than Corinna is needed for this protein injection therapy. Schneider also hopes to organize a clinical trial with the help of the charity foundation, and the treatment team has successfully treated another pregnant woman after the success of the Corinna, but then stopped trying.
it's a pity that this treatment faces another problem, no pharmaceutical company can expect it to make money, and no company will sell it for the drug, even if it works. The reason is that the disease is too rare. Only 1 out of 25000 cases, and the treatment of fetuses in utero will increase the risk of pregnant women dramatically.