Results 191 to 200 of about 284,599 (305)

Integrated Modeling of Digital-Motor and Clinician-Reported Outcomes Using Item Response Theory: Towards Powerful Trials for Rare Neurological Diseases. [PDF]

open access: yesCPT Pharmacometrics Syst Pharmacol
Hamdan A   +16 more
europepmc   +1 more source

A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases. [PDF]

open access: yesNat Commun
Kim-McManus O   +14 more
europepmc   +1 more source

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation. [PDF]

open access: yesMol Ther Nucleic Acids
Schüle R   +6 more
europepmc   +1 more source

Hyperactivity of the non-canonical inflammasome in SPG11 and SPG48. [PDF]

open access: yesEBioMedicine
Afzal MA   +13 more
europepmc   +1 more source

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