Continuous improvement of patient symptoms Parkinson's gene therapy long-term clinical positive.

Parkinson's disease is the second most common neurodegenerative disease after Alzheimer's disease.
the Parkinson's Foundation estimates that there are more than 10 million patients worldwide.
that the disease's motor symptoms are caused by the absence of neurons that produce the neurotransmitter dopamine, substantia nigra.
, LYOD is still the standard treatment for the disease.
Although L-Doba can significantly improve the patient's motor symptoms in the early stages of the disease, as the disease progresses, the patient's response to treatment gradually decreases, and sometimes medication does not achieve optimal results, a period known as the shutdown period ("off" time).
even during the open period of the drug's effects ("on" time), patients may develop dyskinesia (dyskinesia) characterized by involuntary abnormal muscle movement.
Therapeutics is a biotechnology company dedicated to the development of gene therapies to treat neurological diseases.
NBIb-1817 is a genetically modified gene therapy that uses AAV2 virus vectors to carry the expression of the human aromatic L-amino acid dehydrase (AADC).
the characteristics of Parkinson's disease, in addition to the death of black neurons who synthesize dopamine, the level of AADC required to synthesize dopamine in the brain is also declining.
NBIb-1817 is designed to help improve the patient's motor symptoms by expressing AADC enzymes in brain cells to help convert lysodab into dopamine.
this innovative gene therapy has been recognized by the FDA as an advanced therapy for regenerative medicine.
Biosciences, a company, called Neocrine Biosciences, between China and Voyager in January 2019 to develop a number of gene therapies, including NBIb-1817. the goal of
NBIb-1817 gene therapy is to "turn back the clock" and restore symptoms in patients with advanced Parkinson's disease to the level of early patients (Photo Source: Reference: Reference 3) In a Phase 1b open-label clinical trial called PD-1101, 15 patients with moderate to late-stage Parkinson's disease received an NBIb-1817 treatment (divided into three groups, receiving different doses of gene therapy).
results released by the Department of Health showed that after three years of treatment, the patient's closing period decreased by an average of 0.15-1.91 hours (baseline 4.28-4.93 hours).
the length of the open period without movement disorders increased by an average of 0.26-2.23 hours (baseline 10.32-10.46 hours).
, the demand for oral drugs was reduced in patients receiving higher doses of gene therapy in Queue 2 and Queue 3 patients.
the average daily oral dose of L-Doba was reduced by 322.0 and 441.2 mg/day (baselines of 1507.0 and 1477.0 mg/day).
of motor function in patients assessed by clinicians also improved continuously after 3 years.
"The results are very encouraging because the motor function of people with Parkinson's disease usually declines over a three-year period," said lead researcher Dr. Chad Christine, a professor of neurology at the University of California, San Francisco.
" References: s1. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Retrieved September 12, 2020, from s.2. Voyager Therapeutics 2018 Annual Report. Retrieved September 12, 2020, from s.3. Voyager Therapeutics 10K form. Retrieved September 12, 2020, from original title: One treatment, 3 years later continued to improve patient symptoms, Parkinson's disease gene therapy long-term clinical results positive.