Bringing Advanced Therapies for Parkinson's Disease to the Clinic: The Scientist's Perspective
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Bringing Advanced Therapies for Parkinson's Disease to the Clinic : The Scientist's Perspective. / Tomishima, Mark; Kirkeby, Agnete.
In: Journal of Parkinson's Disease, Vol. 11, 2021, p. S135-S140.Research output: Contribution to journal › Review › Research › peer-review
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TY - JOUR
T1 - Bringing Advanced Therapies for Parkinson's Disease to the Clinic
T2 - The Scientist's Perspective
AU - Tomishima, Mark
AU - Kirkeby, Agnete
PY - 2021
Y1 - 2021
N2 - After many years of preclinical development, cell and gene therapies have advanced from research tools in the lab to clinical-grade products for patients, and today they constitute more than a quarter of all new Phase I clinical trials for Parkinson's disease. Whereas efficacy has been convincingly proven for many of these products in preclinical models, the field is now entering a new phase where the functionality and safety of these products will need to stand the test in clinical trials. If successful, these new products can have the potential to provide patients with a one-time administered treatment which may alleviate them from daily symptomatic dopaminergic medication.
AB - After many years of preclinical development, cell and gene therapies have advanced from research tools in the lab to clinical-grade products for patients, and today they constitute more than a quarter of all new Phase I clinical trials for Parkinson's disease. Whereas efficacy has been convincingly proven for many of these products in preclinical models, the field is now entering a new phase where the functionality and safety of these products will need to stand the test in clinical trials. If successful, these new products can have the potential to provide patients with a one-time administered treatment which may alleviate them from daily symptomatic dopaminergic medication.
KW - ATMP
KW - clinical trial
KW - dopaminergic neurons
KW - regenerative therapy
KW - stem cells
KW - transplantation
KW - DOPAMINE NEURONS
KW - PRECLINICAL EFFICACY
KW - CELL TRANSPLANTATION
KW - STEM-CELLS
KW - MODEL
KW - SAFETY
KW - PATHOLOGY
KW - REVEALS
KW - TRIAL
U2 - 10.3233/JPD-212685
DO - 10.3233/JPD-212685
M3 - Review
C2 - 34250954
VL - 11
SP - S135-S140
JO - Journal of Parkinson's Disease
JF - Journal of Parkinson's Disease
SN - 1877-7171
ER -
ID: 281275936