Muscular Atrophy
Recent clinical, regulatory, research and industry developments relating to this disease.
Effects of thyroid hormones in skeletal muscle protein turnover.
Sarcopenia - Molecular mechanisms and open questions.
Mechanisms of IGF-1-Mediated Regulation of Skeletal Muscle Hypertrophy and Atrophy.
Ultrastructural Characterization of the Lower Motor System in a Mouse Model of Krabbe Disease.
Mechanisms regulating skeletal muscle growth and atrophy.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 1 regulatory approval from EMA on record.
- 5 clinical trials expected to report results, the earliest in Q1 2028.
- Q1 2028A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
- Q2 2028An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With Risdiplam
- Q4 2028An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy
- Q4 2030Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
- Q2 2031Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Clinical MilestonesViewHide
- 2026-01-26Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionResults posted
- 2026-01-26Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionResults posted
- 2026-07-09Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical TrialsResults expected Q2 2031
- 2026-07-06A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene TherapyResults expected Q1 2028
- 2026-07-06An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular AtrophyResults expected Q4 2028
- 2026-04-21An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With RisdiplamResults expected Q2 2028
- 2025-12-04Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).Results expected Q4 2030
Regulatory UpdatesViewHide
- 2026-06-30Approval — Onasemnogene abeparvovecItvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older.
- 2026-07-09ClinicalLong-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical TrialsResults expected Q2 2031
- 2026-07-06ClinicalA Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene TherapyResults expected Q1 2028
- 2026-07-06ClinicalAn Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular AtrophyResults expected Q4 2028
- 2026-06-30RegulatoryApproval — Onasemnogene abeparvovecItvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older.
- 2026-04-21ClinicalAn Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With RisdiplamResults expected Q2 2028
- 2026-01-26ClinicalPhase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionResults posted
- 2026-01-26ClinicalPhase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionResults posted
- 2025-12-04ClinicalReal World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).Results expected Q4 2030
- 2025-10-09ClinicalA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecCompleted
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Itvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi-alle… (2026)
Accelerated approval — Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients wi… (2021)
Accelerated approval — Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy. (2017)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Muscle, Skeletal2
- Muscular Atrophy2
- Energy Metabolism1
- Glucagon-Like Peptide-1 Receptor Agonists1
- Health Behavior1
- Hyperthyroidism1
- Muscle Proteins1
- Pandemics1
Leading journals6
- Ageing research reviews1
- Cells1
- European journal of sport science1
- International journal of molecular medicine1
- Journal of basic and clinical physiology and pharmacology1
- Scientific reports1
Leading researchers8
- Bai X1
- Baldassarre G1
- Biolo G1
- Blaauw B1
- Cappello V1
- Castro JP1
- Cecchini M1
- Ciciliot S1
Affiliations (unnormalised)6
- Bispebjerg-Frederiksberg University Hospital1
- Center for Nanotechnology1
- CIR-MYO Myology Center1
- Department of Clinical Medicine and Surgery1
- German Institute of Human Nutrition Potsdam-Rehbrücke1
- Heart and Vascular Institute1
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Muscular atrophy is a reduction in the size and number of muscle fibers. It can occur with aging, reduced blood supply, immobilization, prolonged weightlessness, malnutrition, and especially denervation. The literature also treats it as a muscle-wasting state in which skeletal muscle mass and function decline.
The supplied grounding identifies aging, reduced blood supply, immobilization, prolonged weightlessness, malnutrition, and denervation as causes or triggers. Review material also links muscle wasting to thyroid dysfunction, sepsis-induced myopathy, and age-related sarcopenia as related clinical contexts. These sources support a multifactorial etiology rather than a single cause.
The central biological theme is an imbalance between muscle protein synthesis and protein degradation. Review abstracts describe involvement of IGF-1/Akt/mTOR signaling, FoxO-dependent ubiquitin-proteasome pathways, myostatin-Smad signaling, inflammatory pathways, and autophagy-related mechanisms. Changes in satellite cell activity, proteostasis, mitochondrial function, and neuromuscular drive are also described in the literature.
Aging is a major risk factor, and the grounding also supports immobilization, prolonged weightlessness, malnutrition, denervation, and reduced blood supply. Related literature additionally associates muscle wasting with thyroid dysfunction and sepsis. Sarcopenia is described as an age-related syndrome with higher risk of falls, fractures, metabolic disease, and mortality, but those are consequences rather than primary risk factors.
The grounding does not support a single established standard treatment for muscular atrophy. The literature emphasizes prevention and modulation of underlying pathways, with drug-therapy discussions centered on anabolic or anti-catabolic signaling such as IGF-1-related pathways, myostatin inhibition, and GLP-1 receptor agonists in sepsis-induced muscle atrophy. At the modality level, strength exercise and contractile activity are described as stimuli that can promote muscle hypertrophy rather than atrophy.
AI-generated summary grounded in MeSH and 5 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Derangement in size and number of muscle fibers occurring with aging, reduction in blood supply, or following immobilization, prolonged weightlessness, malnutrition, and particularly in denervation.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.