Platform trial design for neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis: A potential model for rare diseases.
Dhaenens BAE, Heimann G, Bakker A, Nievo M, Ferner RE, Evans DG, Wolkenstein P, Leubner J, Potratz C, Carton C, Iloeje U, Kirk G, Blakeley JO, Plotkin S, Fisher MJ, Kim A, Driever PH, Azizi AA, Widemann BC, Gross A, Parke T, Legius E, Oostenbrink R.
Neurooncol Pract. 2024 Jan 4;11(4):395-403. doi: 10.1093/nop/npae001. eCollection 2024 Aug.
PMID:39006526
The case for not masking away repetitive DNA.
Slotkin RK.
Mob DNA. 2018 May 1;9:15. doi: 10.1186/s13100-018-0120-9. eCollection 2018.
PMID:29743957
Modeling of Parkinson's Disease Progression and Implications for Detection of Disease Modification in Treatment Trials.
Ribba B, Simuni T, Marek K, Siderowf A, Diack C, Pierrillas PB, Monnet A, Ricci B, Nikolcheva T, Pagano G.