1. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy. (24th October 2017) Authors: Victor, Ronald G.; Sweeney, H. Lee; Finkel, Richard; McDonald, Craig M.; Byrne, Barry; Eagle, Michelle; Goemans, Nathalie; Vandenborne, Krista; Dubrovsky, Alberto L.; Topaloglu, Haluk; Miceli, M. Carrie; Furlong, Pat; Landry, John; Elashoff, Robert; Cox, David Journal: Neurology Issue: Volume 89:Number 17(2017) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Issue 4 (October 2014) Authors: Bushby, Katharine; Finkel, Richard; Wong, Brenda; Barohn, Richard; Campbell, Craig; Comi, Giacomo P.; Connolly, Anne M.; Day, John W.; Flanigan, Kevin M.; Goemans, Nathalie; Jones, Kristi J.; Mercuri, Eugenio; Quinlivan, Ros; Renfroe, James B.; Russman, Barry; Ryan, Monique M.; Tulinius, Mar; Voi... Journal: Muscle & nerve Issue: Volume 50:Issue 4(2014:Oct.) Page Start: 477 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Issue 2 (21st January 2016) Authors: Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Iannaccone, Susan T.; Parsons, Julie; Connolly, Anne M.; Chiribog... Journal: Annals of clinical and translational neurology Issue: Volume 3:Issue 2(2016) Page Start: 132 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Different trajectories in upper limb and gross motor function in spinal muscular atrophy. Issue 5 (9th August 2021) Authors: Coratti, Giorgia; Pera, Maria Carmela; Montes, Jacqueline; Pasternak, Amy; Scoto, Mariacristina; Baranello, Giovanni; Messina, Sonia; Dunaway Young, Sally; Glanzman, Allan M.; Duong, Tina; De Sanctis, Roberto; Mazzone, Elena Stacy; Milev, Evelin; Rohwer, Annemarie; Civitello, Matthew; Pane, Marik... Journal: Muscle & nerve Issue: Volume 64:Issue 5(2021) Page Start: 552 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Evolution of Next Generation Therapeutics: Past, Present, and Future of Precision Medicines. Issue 6 (23rd August 2019) Authors: Ramamoorthy, Anuradha; Karnes, Jason H.; Finkel, Richard; Blanchard, Rebecca; Pacanowski, Michael Journal: Clinical and translational science Issue: Volume 12:Issue 6(2019) Page Start: 560 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Genetic therapies for inherited neuromuscular disorders. (August 2018) Authors: Scoto, Mariacristina; Finkel, Richard; Mercuri, Eugenio; Muntoni, Francesco Journal: Lancet Issue: Volume 2:Number 8(2018) Page Start: 600 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy. Issue 4 (7th March 2022) Authors: Waldrop, Megan A.; Moore, Steven A.; Mathews, Katherine D.; Darbro, Benjamin W.; Medne, Livja; Finkel, Richard; Connolly, Anne M.; Crawford, Thomas O.; Drachman, Daniel; Wein, Nicolas; Habib, Ali A.; Krzesniak‐Swinarska, Monika A.; Zaidman, Craig M.; Collins, James J.; Jokela, Manu; Udd, Bjarne; ... Journal: Human mutation Issue: Volume 43:Issue 4(2022) Page Start: 511 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Modeling disease trajectory in Duchenne muscular dystrophy. (14th April 2020) Authors: Rooney, William D.; Berlow, Yosef A.; Triplett, William T.; Forbes, Sean C.; Willcocks, Rebecca J.; Wang, Dah-Jyuu; Arpan, Ishu; Arora, Harneet; Senesac, Claudia; Lott, Donovan J.; Tennekoon, Gihan; Finkel, Richard; Russman, Barry S.; Finanger, Erika L.; Chakraborty, Saptarshi; O'Brien, Elliott; ... Journal: Neurology Issue: Volume 94:Number 15(2020) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. Natural history of infantile‐onset spinal muscular atrophy. Issue 6 (8th December 2017) Authors: Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Parsons, Julie; Connolly, Anne M.; Chiriboga, Claudia A.; McDonal... Journal: Annals of neurology Issue: Volume 82:Issue 6(2017) Page Start: 883 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
10. Nusinersen in pediatric and adult patients with type III spinal muscular atrophy. Issue 8 (24th June 2021) Authors: Pera, Maria Carmela; Coratti, Giorgia; Bovis, Francesca; Pane, Marika; Pasternak, Amy; Montes, Jacqueline; Sansone, Valeria A.; Dunaway Young, Sally; Duong, Tina; Messina, Sonia; Mizzoni, Irene; D'Amico, Adele; Civitello, Matthew; Glanzman, Allan M.; Bruno, Claudio; Salmin, Francesca; Morando, Si... Journal: Annals of clinical and translational neurology Issue: Volume 8:Issue 8(2021) Page Start: 1622 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗