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]
Hamdan A +16 more
europepmc +1 more source
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases. [PDF]
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]
Schüle R +6 more
europepmc +1 more source
Hyperactivity of the non-canonical inflammasome in SPG11 and SPG48. [PDF]
Afzal MA +13 more
europepmc +1 more source

