Therapy for Spinal Muscular Atrophy

Heather T Whittaker^{1

2}, Mackenzie A Michell-Robinson¹

Affiliations

Comment

Heather T Whittaker et al. N Engl J Med. 2018.

. 2018 Feb 1;378(5):487.

doi: 10.1056/NEJMc1715769.

Heather T Whittaker^{1

2}, Mackenzie A Michell-Robinson¹

No abstract available

Comment in

Therapy for Spinal Muscular Atrophy.
Finkel RS, Farwell W. Finkel RS, et al. N Engl J Med. 2018 Feb 1;378(5):487-488. doi: 10.1056/NEJMc1715769. N Engl J Med. 2018. PMID: 29385371 No abstract available.
Therapy for Spinal Muscular Atrophy.
Mendell JR. Mendell JR. N Engl J Med. 2018 Feb 1;378(5):487. doi: 10.1056/NEJMc1715769. N Engl J Med. 2018. PMID: 29394473 No abstract available.

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Clinical Trial.
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Finkel RS, et al. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Clinical Trial.