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→SMN2 alternative splicing modulation: I worked on the pre-IND sent to the FDA and it was in 2013 not 2010, shortly thereafter partake downsized and concentrated on its tetracycline antibiotic on what is now NUZYRA, which was approved by the FDA in 2019 |
→Neuroprotection: Add GYM329; minor copyedits elsewhere. |
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[[Neuroprotection|Neuroprotective]] drugs aim at enabling the survival of motor neurons even with low levels of SMN protein.
* [[Olesoxime]] was a proprietary neuroprotective compound developed by the French company [[Trophos]], later acquired by [[Hoffmann-La Roche]], which showed stabilising effect in a phase-II clinical trial involving people with SMA types 2 and 3. Its development was discontinued in 2018 in view of competition
Of clinically studied compounds which did not show efficacy, [[thyrotropin-releasing hormone]] (TRH) held some promise in an [[open-label trial|open-label]] [[uncontrolled trial|uncontrolled]] clinical trial<ref>{{cite journal | vauthors = Takeuchi Y, Miyanomae Y, Komatsu H, Oomizono Y, Nishimura A, Okano S, Nishiki T, Sawada T | title = Efficacy of thyrotropin-releasing hormone in the treatment of spinal muscular atrophy | journal = Journal of Child Neurology | volume = 9 | issue = 3 | pages = 287–9 | date = July 1994 | pmid = 7930408 | doi = 10.1177/088307389400900313 | s2cid = 41678161 }}</ref><ref>{{cite journal | vauthors = Tzeng AC, Cheng J, Fryczynski H, Niranjan V, Stitik T, Sial A, Takeuchi Y, Foye P, DePrince M, Bach JR | title = A study of thyrotropin-releasing hormone for the treatment of spinal muscular atrophy: a preliminary report | journal = American Journal of Physical Medicine & Rehabilitation | volume = 79 | issue = 5 | pages = 435–40 | year = 2000 | pmid = 10994885 | doi = 10.1097/00002060-200009000-00005 | s2cid = 20416253 }}</ref><ref>{{cite journal | vauthors = Kato Z, Okuda M, Okumura Y, Arai T, Teramoto T, Nishimura M, Kaneko H, Kondo N | title = Oral administration of the thyrotropin-releasing hormone (TRH) analogue, taltireline hydrate, in spinal muscular atrophy | journal = Journal of Child Neurology | volume = 24 | issue = 8 | pages = 1010–2 | date = August 2009 | pmid = 19666885 | doi = 10.1177/0883073809333535 | s2cid = 29321906 }}</ref> but did not prove effective in a subsequent [[double-blind trial|double-blind]] [[placebo-controlled]] trial.<ref>{{cite journal | vauthors = Wadman RI, Bosboom WM, van den Berg LH, Wokke LH, Iannaccone ST, Vrancken AF |editor1-first =Renske I |editor1-last =Wadman |title =Drug treatment for spinal muscular atrophy type I |date=2011-12-07 | collaboration = The Cochrane Collaboration |publisher=John Wiley & Sons, Ltd |doi=10.1002/14651858.cd006281.pub3 |journal =Cochrane Database of Systematic Reviews |issue =12 |pages =CD006281 |pmid =22161399 }}</ref> [[Riluzole]], a drug that
=== Muscle restoration ===
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This approach aims to counter the effect of SMA by targeting the muscle tissue instead of neurons.
* Reldesemtiv (CK-2127107, CK-107) is a skeletal [[troponin]] activator developed by Cytokinetics in cooperation with [[Astellas]]. The drug aims at increasing muscle reactivity despite lowered neural signalling. The molecule showed some success in phase II clinical trial in adolescent and adults with SMA types 2, 3, and 4.<ref>{{cite web | url=http://cytokinetics.com/ck-2127107|title=CK-2127107 }}</ref>
* Apitegromab (SRK-015) is [[monoclonal antibody]] that
* GYM329 (RO7204239), developed by Hoffman-La Roche, works similarly to apitegromab by blocking myostatin activation. As of 2022, combined with risdiplam, it is undergoing clinical development in non-ambulant children with SMA aged 2–10.<ref>{{Cite web|last=PhD|first=Patricia Inacio|title=Pediatric Phase 2/3 Trial to Test Anti-myostatin Antibody with Evrysdi|url=https://smanewstoday.com/news-posts/2021/10/25/pediatric-phase-2-3-trial-test-anti-myostatin-antibody-gym329-with-evrysdi/|access-date=2022-01-23|language=en-US}}</ref>
===Stem cells===
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