On Tuesday, Bethesda Children's Hospital introduced an innovative, one-time gene therapy drug to Sentinel, a second-year patient with SMA1.As early as Wednesday, Zente can leave the hospital after receiving treatment (Photo: Vargos). Gene therapy, patented in the United States and not yet licensed in Europe, has been used on three continents to date: this one-time treatment can reduce the burden of disease on the patient, the family, and the health system, replacing repetitive, lifelong therapy. At the request of the parents, Bethesda Children's Hospital, as the national SMA center, has completed treatment preparation, procurement logistics and drug delivery, which was successfully completed on October 29th.Zente was well on her way to the medication and is currently in a segregated environment to expect to return home the day after the medication. SMA (spinal muscular atrophy) is a rare genetic disorder characterized by the loss of function of the nerve cells in the spinal cord that move the musculoskeletal system, leading to weakness, loss of muscle movements and loss of muscle without treatment. The cure for the disease is a unique, high-value treatment available with two drugs - but the evidence for its long-term effect is still limited. Zente is in the care of the Bethesda Children's Hospital, where the national SMA center is treated intrathecally, that is to say, by a single injection of a spinal cord. The condition of the child has significantly improved with the effect of a drug introduced in Hungary one year ago. Injections cost 130 million forints in the first year, and 67.5 million forints each year for sustained practitioners, and are funded by the Hungarian Social Security. This drug has been given to around 8,000 children worldwide, so it has much more experience with its effectiveness. In their international parenting, the baby boomers have reported the availability of three new gene therapy therapies made in the US and used in Europe up to three patients, which have been received by about 100 children worldwide. They collect the amount needed for purchasing, shipping and preparing medicines by social gathering, And, after all the precautions, it was referred to the House of Culture. szьksйges the gyуgyszer beadбsбhoz professional elхkйszьleteket vйgezte the kуrhбz to which the gyбrtуtуl the Egyesьlt Бllamokbуl йrkezett szakйrtх: х speciбlis oktatбst held kezelйst vйgzх munkatбrsak kцrйben the mыszereket the kуrtermeket and this megfelelхen kйszнtettйk elх the kуrhбzban, everything garantбlva the largest biztonsбgot. According to the hospital's announcement, the preparation of the patient, the administrative organization of the medication, the transfer, and the creation of the administrative, financial and legal conditions were all well done. The drug arrived in the Bethesda Children's Hospital Institutional Pharmacy on October 28, according to prescriptions. Because the invention requires innovative, extreme conditions, the hospital needed the attention of many of its employees to coordinate its treatment at the right time. Our previous articles in this topic:
- Zente received the handler on Tuesday
- Zente therapy is about to begin
- He managed to raise 700 million for Zente
- Call for help Levi heal together!