Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy Article Swipe
Laurent Servais
,
Lindsey Lair
,
Anne M. Connolly
,
Barry J. Byrne
,
Karen Chen
,
Vlad Coric
,
Irfan Qureshi
,
Susan Durham
,
Daniel Campbell
,
Grant Maclaine
,
Jackie Marin
,
Clifford Bechtold
·
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.3390/ijms251910273
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.3390/ijms251910273
Spinal muscular atrophy (SMA) is a rare, genetic neurodegenerative disorder caused by insufficient production of survival motor neuron (SMN) protein. Diminished SMN protein levels lead to motor neuron loss, causing muscle atrophy and weakness that impairs daily functioning and reduces quality of life. SMN upregulators offer clinical improvements and increased survival in SMA patients, although significant unmet needs remain. Myostatin, a TGF-β superfamily signaling molecule that binds to the activin II receptor, negatively regulates muscle growth; myostatin inhibition is a promising therapeutic strategy for enhancing muscle. Combining myostatin inhibition with SMN upregulation, a comprehensive therapeutic strategy targeting the whole motor unit, offers promise in SMA. Taldefgrobep alfa is a novel, fully human recombinant protein that selectively binds to myostatin and competitively inhibits other ligands that signal through the activin II receptor. Given a robust scientific and clinical rationale and the favorable safety profile of taldefgrobep in patients with neuromuscular disease, the RESILIENT phase 3, randomized, placebo-controlled trial is investigating taldefgrobep as an adjunct to SMN upregulators in SMA (NCT05337553). This manuscript reviews the role of myostatin in muscle, explores the preclinical and clinical development of taldefgrobep and introduces the phase 3 RESILIENT trial of taldefgrobep in SMA.
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Metadata
- Type
- review
- Language
- en
- Landing Page
- https://doi.org/10.3390/ijms251910273
- OA Status
- gold
- Cited By
- 9
- References
- 81
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4402761078
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
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https://openalex.org/W4402761078Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.3390/ijms251910273Digital Object Identifier
- Title
-
Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular AtrophyWork title
- Type
-
reviewOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2024Year of publication
- Publication date
-
2024-09-24Full publication date if available
- Authors
-
Laurent Servais, Lindsey Lair, Anne M. Connolly, Barry J. Byrne, Karen Chen, Vlad Coric, Irfan Qureshi, Susan Durham, Daniel Campbell, Grant Maclaine, Jackie Marin, Clifford BechtoldList of authors in order
- Landing page
-
https://doi.org/10.3390/ijms251910273Publisher landing page
- Open access
-
YesWhether a free full text is available
- OA status
-
goldOpen access status per OpenAlex
- OA URL
-
https://doi.org/10.3390/ijms251910273Direct OA link when available
- Concepts
-
Myostatin, Spinal muscular atrophy, SMA*, Activin receptor, Motor neuron, Medicine, Neuroscience, Weakness, Internal medicine, Bioinformatics, Receptor, Biology, Disease, Skeletal muscle, Anatomy, Computer science, AlgorithmTop concepts (fields/topics) attached by OpenAlex
- Cited by
-
9Total citation count in OpenAlex
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2025: 9Per-year citation counts (last 5 years)
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81Number of works referenced by this work
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10Other works algorithmically related by OpenAlex
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| referenced_works | https://openalex.org/W4382599512, https://openalex.org/W4289755145, https://openalex.org/W2783169765, https://openalex.org/W4387306320, https://openalex.org/W3113750725, https://openalex.org/W4387729616, https://openalex.org/W2765483614, https://openalex.org/W2765253110, https://openalex.org/W3186720955, https://openalex.org/W4200042237, https://openalex.org/W3160334825, https://openalex.org/W3135935053, https://openalex.org/W3206999035, https://openalex.org/W2789114047, https://openalex.org/W2560202024, https://openalex.org/W4285589111, https://openalex.org/W2987746715, https://openalex.org/W3004089807, https://openalex.org/W4308425595, https://openalex.org/W4391589874, https://openalex.org/W4213113433, https://openalex.org/W2166877291, https://openalex.org/W4283213615, https://openalex.org/W2165868509, https://openalex.org/W2053079324, https://openalex.org/W3154035165, https://openalex.org/W2951326659, https://openalex.org/W3199536016, https://openalex.org/W4211003966, https://openalex.org/W4283320528, https://openalex.org/W2770028464, https://openalex.org/W4229074058, https://openalex.org/W4223971949, https://openalex.org/W2166485013, https://openalex.org/W4390663037, https://openalex.org/W4386916958, https://openalex.org/W2102450141, https://openalex.org/W2071655559, https://openalex.org/W2912779195, https://openalex.org/W4401591205, https://openalex.org/W3005103172, https://openalex.org/W3135290722, https://openalex.org/W4394599565, https://openalex.org/W2609426395, https://openalex.org/W2607657850, https://openalex.org/W2158739907, https://openalex.org/W3087490549, https://openalex.org/W3082888217, https://openalex.org/W4205657977, https://openalex.org/W2082994316, https://openalex.org/W1978567675, https://openalex.org/W3197221403, https://openalex.org/W4307106827, https://openalex.org/W2913924970, https://openalex.org/W3084730147, https://openalex.org/W2589203314, https://openalex.org/W4318457111, https://openalex.org/W4312060028, https://openalex.org/W2528264181, https://openalex.org/W3212347834, https://openalex.org/W4200142199, https://openalex.org/W2322137887, https://openalex.org/W3133963998, https://openalex.org/W2172191780, https://openalex.org/W3094596692, https://openalex.org/W2784992484, https://openalex.org/W2984099159, https://openalex.org/W4385278403, https://openalex.org/W3136414815, https://openalex.org/W2114859624, https://openalex.org/W4319058509, https://openalex.org/W4307313911, https://openalex.org/W4317394414, https://openalex.org/W4317499915, https://openalex.org/W3191422445, https://openalex.org/W4387451497, https://openalex.org/W4380052062, https://openalex.org/W4281694433, https://openalex.org/W4394579473, https://openalex.org/W4394579849, https://openalex.org/W3140066921 |
| referenced_works_count | 81 |
| abstract_inverted_index.3 | 190 |
| abstract_inverted_index.a | 5, 60, 79, 92, 108, 132 |
| abstract_inverted_index.3, | 153 |
| abstract_inverted_index.II | 70, 129 |
| abstract_inverted_index.an | 161 |
| abstract_inverted_index.as | 160 |
| abstract_inverted_index.by | 11 |
| abstract_inverted_index.in | 51, 103, 145, 166, 176, 195 |
| abstract_inverted_index.is | 4, 78, 107, 157 |
| abstract_inverted_index.of | 14, 41, 143, 174, 184, 193 |
| abstract_inverted_index.to | 25, 67, 117, 163 |
| abstract_inverted_index.SMA | 52, 167 |
| abstract_inverted_index.SMN | 21, 43, 90, 164 |
| abstract_inverted_index.and | 32, 38, 48, 119, 135, 138, 181, 186 |
| abstract_inverted_index.for | 83 |
| abstract_inverted_index.the | 68, 97, 127, 139, 150, 172, 179, 188 |
| abstract_inverted_index.SMA. | 104, 196 |
| abstract_inverted_index.This | 169 |
| abstract_inverted_index.alfa | 106 |
| abstract_inverted_index.lead | 24 |
| abstract_inverted_index.role | 173 |
| abstract_inverted_index.that | 34, 65, 114, 124 |
| abstract_inverted_index.with | 89, 147 |
| abstract_inverted_index.(SMA) | 3 |
| abstract_inverted_index.(SMN) | 18 |
| abstract_inverted_index.Given | 131 |
| abstract_inverted_index.binds | 66, 116 |
| abstract_inverted_index.daily | 36 |
| abstract_inverted_index.fully | 110 |
| abstract_inverted_index.human | 111 |
| abstract_inverted_index.life. | 42 |
| abstract_inverted_index.loss, | 28 |
| abstract_inverted_index.motor | 16, 26, 99 |
| abstract_inverted_index.needs | 57 |
| abstract_inverted_index.offer | 45 |
| abstract_inverted_index.other | 122 |
| abstract_inverted_index.phase | 152, 189 |
| abstract_inverted_index.rare, | 6 |
| abstract_inverted_index.trial | 156, 192 |
| abstract_inverted_index.unit, | 100 |
| abstract_inverted_index.unmet | 56 |
| abstract_inverted_index.whole | 98 |
| abstract_inverted_index.Spinal | 0 |
| abstract_inverted_index.TGF-β | 61 |
| abstract_inverted_index.caused | 10 |
| abstract_inverted_index.levels | 23 |
| abstract_inverted_index.muscle | 30, 74 |
| abstract_inverted_index.neuron | 17, 27 |
| abstract_inverted_index.novel, | 109 |
| abstract_inverted_index.offers | 101 |
| abstract_inverted_index.robust | 133 |
| abstract_inverted_index.safety | 141 |
| abstract_inverted_index.signal | 125 |
| abstract_inverted_index.activin | 69, 128 |
| abstract_inverted_index.adjunct | 162 |
| abstract_inverted_index.atrophy | 2, 31 |
| abstract_inverted_index.causing | 29 |
| abstract_inverted_index.genetic | 7 |
| abstract_inverted_index.growth; | 75 |
| abstract_inverted_index.impairs | 35 |
| abstract_inverted_index.ligands | 123 |
| abstract_inverted_index.muscle, | 177 |
| abstract_inverted_index.muscle. | 85 |
| abstract_inverted_index.profile | 142 |
| abstract_inverted_index.promise | 102 |
| abstract_inverted_index.protein | 22, 113 |
| abstract_inverted_index.quality | 40 |
| abstract_inverted_index.reduces | 39 |
| abstract_inverted_index.remain. | 58 |
| abstract_inverted_index.reviews | 171 |
| abstract_inverted_index.through | 126 |
| abstract_inverted_index.although | 54 |
| abstract_inverted_index.clinical | 46, 136, 182 |
| abstract_inverted_index.disease, | 149 |
| abstract_inverted_index.disorder | 9 |
| abstract_inverted_index.explores | 178 |
| abstract_inverted_index.inhibits | 121 |
| abstract_inverted_index.molecule | 64 |
| abstract_inverted_index.muscular | 1 |
| abstract_inverted_index.patients | 146 |
| abstract_inverted_index.protein. | 19 |
| abstract_inverted_index.strategy | 82, 95 |
| abstract_inverted_index.survival | 15, 50 |
| abstract_inverted_index.weakness | 33 |
| abstract_inverted_index.Combining | 86 |
| abstract_inverted_index.RESILIENT | 151, 191 |
| abstract_inverted_index.enhancing | 84 |
| abstract_inverted_index.favorable | 140 |
| abstract_inverted_index.increased | 49 |
| abstract_inverted_index.myostatin | 76, 87, 118, 175 |
| abstract_inverted_index.patients, | 53 |
| abstract_inverted_index.promising | 80 |
| abstract_inverted_index.rationale | 137 |
| abstract_inverted_index.receptor, | 71 |
| abstract_inverted_index.receptor. | 130 |
| abstract_inverted_index.regulates | 73 |
| abstract_inverted_index.signaling | 63 |
| abstract_inverted_index.targeting | 96 |
| abstract_inverted_index.Diminished | 20 |
| abstract_inverted_index.Myostatin, | 59 |
| abstract_inverted_index.inhibition | 77, 88 |
| abstract_inverted_index.introduces | 187 |
| abstract_inverted_index.manuscript | 170 |
| abstract_inverted_index.negatively | 72 |
| abstract_inverted_index.production | 13 |
| abstract_inverted_index.scientific | 134 |
| abstract_inverted_index.development | 183 |
| abstract_inverted_index.functioning | 37 |
| abstract_inverted_index.preclinical | 180 |
| abstract_inverted_index.randomized, | 154 |
| abstract_inverted_index.recombinant | 112 |
| abstract_inverted_index.selectively | 115 |
| abstract_inverted_index.significant | 55 |
| abstract_inverted_index.superfamily | 62 |
| abstract_inverted_index.therapeutic | 81, 94 |
| abstract_inverted_index.Taldefgrobep | 105 |
| abstract_inverted_index.improvements | 47 |
| abstract_inverted_index.insufficient | 12 |
| abstract_inverted_index.taldefgrobep | 144, 159, 185, 194 |
| abstract_inverted_index.upregulators | 44, 165 |
| abstract_inverted_index.competitively | 120 |
| abstract_inverted_index.comprehensive | 93 |
| abstract_inverted_index.investigating | 158 |
| abstract_inverted_index.neuromuscular | 148 |
| abstract_inverted_index.upregulation, | 91 |
| abstract_inverted_index.(NCT05337553). | 168 |
| abstract_inverted_index.neurodegenerative | 8 |
| abstract_inverted_index.placebo-controlled | 155 |
| cited_by_percentile_year.max | 99 |
| cited_by_percentile_year.min | 98 |
| countries_distinct_count | 3 |
| institutions_distinct_count | 12 |
| citation_normalized_percentile.value | 0.96159635 |
| citation_normalized_percentile.is_in_top_1_percent | False |
| citation_normalized_percentile.is_in_top_10_percent | True |