Hemophilia is a rare disorder in which the blood doesn't clot properly. Following disappointing results in its gene therapy trial for haemophilia, Pfizer -based Spark Therapeutics's shares have fallen by 97% . Spark claims that the experimental treatment for haemophilia A reduces the need for infusions by 97% in a trial of two patients. However, in the higher dosage trial intended for later stage testing, the treatment had an negative reaction in 12 of two patients, with one admitted to hospital with a serious adverse effect.
Following disappointing results in its gene therapy trial for haemophilia, Pfizer -based Spark Therapeutics's shares have fallen by 97% . Spark claims that the experimental treatment for haemophilia A reduces the need for infusions by 97% in a trial of two patients. However, in the higher dosage trial intended for later stage testing, the treatment had an negative reaction in 12 of two patients, with one admitted to hospital with a serious adverse effect.