Atrophy
Recent clinical, regulatory, research and industry developments relating to this disease.
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Lifespan neurodegeneration of the human brain in multiple sclerosis.
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.
- 4 clinical trials expected to report results, the earliest in Q4 2027.
- 2 industry developments reported.
- Q4 2027A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
- Q1 2028Interventional, Randomized, Double-blind, Placebo-controlled, Optional Open-label Extension Trial of Lu AF82422 in Participants With Multiple System Atrophy
- 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
Clinical MilestonesViewHide
- 2026-07-14Interventional, Randomized, Double-blind, Placebo-controlled, Optional Open-label Extension Trial of Lu AF82422 in Participants With Multiple System AtrophyResults expected Q1 2028
- 2026-07-09A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
- 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
- 2026-01-01A Phase 3, Multi-center, Randomized Withdrawal and Long Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants With Multiple System AtrophyPrimary completion
- 2025-10-09A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecCompleted
- 2025-09-05A Phase 3, Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in Subjects With Geographic Atrophy Secondary to Age-Related Macular DegenerationCompleted
Industry & MarketViewHide
- 2026-02-04Meiragtx licenses Zipbio’s gene therapies for geographic atrophyPartnership · BioWorld Gene Therapy
- 2026-05-13BTX-001 enables single-dose treatment for geographic atrophyIndustry · BioWorld Gene Therapy
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.
- 2026-07-14ClinicalInterventional, Randomized, Double-blind, Placebo-controlled, Optional Open-label Extension Trial of Lu AF82422 in Participants With Multiple System AtrophyResults expected Q1 2028
- 2026-07-09ClinicalA Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
- 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.
- 2026-05-18ClinicalAn Open-label Extension (OLE) Phase 3 Trial to Assess the Safety of Intravitreal Administration of Avacincaptad Pegol (Complement C5 Inhibitor) in Patients With Geographic Atrophy Who Previously Completed Phase 3 Study ISEE2008 (GATHER2)Results posted
- 2026-05-13IndustryBTX-001 enables single-dose treatment for geographic atrophyIndustry · BioWorld Gene Therapy
- 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
- 2026-02-04IndustryMeiragtx licenses Zipbio’s gene therapies for geographic atrophyPartnership · BioWorld Gene Therapy
- 2026-01-01ClinicalA Phase 3, Multi-center, Randomized Withdrawal and Long Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants With Multiple System AtrophyPrimary completion
- 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)
Approval — Intrarosa is indicated for the treatment of vulvar and vaginal atrophy in postmenopausal… (2018)
Accelerated approval — Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy. (2017)
Approval — Senshio is indicated for the treatment of moderate to severe symptomatic vulvar and vagin… (2015)
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
- Alzheimer Disease7
- Cognitive Dysfunction5
- Biomarkers2
- Brain2
- Disease Progression2
- Magnetic Resonance Imaging2
- Aging1
- Atrophy1
Leading journals6
- Alzheimer's & dementia : the journal of the Alzheimer's Association3
- Nature communications2
- The Journal of neuroscience : the official journal of the Society for Neuroscience2
- Annals of neurology1
- BMC medicine1
- Brain : a journal of neurology1
Leading researchers8
- Blennow K2
- Karikari TK2
- Zetterberg H2
- Aarsland D1
- Albert NL1
- Altmann A1
- Ashton NJ1
- Baker SL1
Affiliations (unnormalised)6
- Clinical Neurochemistry Laboratory2
- Hong Kong Center for Neurodegenerative Diseases2
- Institute of Neuroscience and Physiology2
- UCL Institute of Neurology2
- UK Dementia Research Institute at UCL2
- University of Pittsburgh2
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Atrophy is a decrease in the size of a cell, tissue, organ, or multiple organs. It is described as occurring in association with a range of pathological conditions, including abnormal cellular changes, ischemia, malnutrition, and hormonal changes.
The supplied grounding supports several associated causes or contexts for atrophy, including abnormal cellular changes, ischemia, malnutrition, and hormonal changes. It also supports disease-specific examples in which atrophy is part of the pathology, such as multifidus dysfunction in chronic low back pain and neurodegenerative conditions discussed in relation to amyloid beta-peptides, tau proteins, and neurofilament proteins.
Atrophy reflects a pathological reduction in tissue or organ size, and the grounding links this to abnormal cellular changes, ischemic injury, malnutrition, and hormonal influences. In the reviewed literature, atrophy is also used as a pathological marker in multifidus dysfunction and in neurodegenerative contexts where structural loss is assessed alongside proteins such as amyloid beta-peptides, tau proteins, and neurofilament proteins.
The grounding supports malnutrition, ischemia, and hormonal changes as factors associated with atrophy. It also indicates that aging is a co-studied mechanism in the literature, suggesting it is relevant to atrophic pathology in some contexts.
The supplied grounding does not support a general standard-of-care treatment for atrophy as a standalone condition. In the reviewed literature, management is context-specific and includes restorative neurostimulation for multifidus dysfunction, while other disease-specific therapies are discussed for underlying disorders rather than for atrophy itself.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Decrease in the size of a cell, tissue, organ, or multiple organs, associated with a variety of pathological conditions such as abnormal cellular changes, ischemia, malnutrition, or hormonal changes.
- 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.