Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

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Sumary of Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients:

  • The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval..
  • Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects profile..
  • They report in the Journal of Neuromuscular Diseases that the drug is associated with an immune response against the adeno-associated viral vector and needs careful monitoring, but showed no long-term adverse effects..
  • One of these is onasemnogene abeparvovec (Zolgensma®), an adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy of the SMN1 gene into motor neurons by means of a single intravenous injection..
  • In the main clinical study on which approval of onasemnogene abeparvovec was based, just 22 babies were given this therapy..
  • Twenty of these were alive and breathing without a permanent ventilator after 14 months, when normally only a quarter of untreated patients would survive without needing a ventilator..
  • Based on these results, the US Food and Drug Administration (FDA) approved the therapy for all types of SMA up to the age of two years, and the European Medicine Agency (EMA) extended the label to all patients either showing a phenotype of SMA type 1 or having up to three SMN2 copies..
  • “However, FDA and EMA approved the treatment for a broader spectrum of patients, which has resulted in discussions about how safe and effective the treatment is in older and heavier patients and in those with SMA type 2.”.
  • Investigators report their experience with eight consecutive patients with SMA who were treated with the standard dose of onasemnogene abeparvovec (1.1×1014 vg/kg) at the University Hospital Bonn, Germany..
  • All patients received prophylactic immunosuppression with prednisolone for four weeks starting on the day before gene therapy..
  • All patients had two or three copies of the SMN2 gene and had been previously treated with nusinersen, also approved for treatment of SMA..
  • Related Stories Following treatment, all of the patients showed a temporary increase of body temperature and an increase of transaminase levels (transaminases are enzymes that are important in the synthesis of amino acids, which form proteins)..
  • Liver values and blood counts returned to normal or almost normal levels during the post-treatment observation period..
  • Four patients had an increase in troponin I levels, which can be a sign of cardiac injury, but cardiac evaluation showed no abnormalities…

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