Spinális izomatrófia (SMA): ki dolgozik rajta

Intézményenként egy kártya. Célpontra vagy országra szűkítheted a listát.

• Minden célpont
• SMN1 4
• VEGF 0
• PARP 0
• TP53 0
• AR 0
• CFTR 1
• DMD 1
• G6PD 0
• HDAC 0
• HTT 1
• NF1 1
• NRF2 0

378 intézményből 24, a friss publikációk és a nyitott vizsgálatok szerint rendezve. Ez nem minőségi rangsor.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  Multi-Material Additive Manufacturing of S690/Fe-Mn-Si-Cr-Ni SMA Joints with 30% Enhanced Static and Fatigue Life for Structural Applications through L-DED (MultiMAMSS)

  Politecnico di Milano· Milano, Olaszország· EU contribution 209 483 EUR· until 2028. márc.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-GF

  Mechanisms of Skeletal Muscle Adaptation to Resistance Training in Spinal Muscular Atrophy (SMA-RT)

  Universita Degli Studi di Padova· Padova, Olaszország· EU contribution 396 991 EUR· until 2029. jan.· 2 countries

• European Commission (Horizon Europe) · HORIZON-ERC-SYG

  Harnessing the splicing code for targeted control of gene expression (UNLEASH)

  Fundacio Centre de Regulacio Genomica· Barcelona, Spanyolország· EU contribution 5 000 764 EUR· until 2029. máj.· 3 countries

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon 2020) · ERC-COG

  Generation of Position Specific organoids to study human neuromuscular system development and disease (GPSorganoids)

  Max Delbrueck Centrum Fuer Molekulare Medizin in Der Helmholtz-Gemeinschaft (Mdc)· Berlin, Németország· EU contribution 2 799 375 EUR· until 2027. dec.

  The disease is named only in the funder's project summary, not in the title.

4 of 4 running projects, newest first. Listed amounts together: 8,4 M EUR.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Leállított, visszavont és felfüggesztett vizsgálatok, a szponzor által a regiszterben megadott okkal, szó szerint idézve.

• Visszavonták

  Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study

  NCT05839145· Institut de Myologie, France· Frissítve: 2026. nov. 4.

  “funding issues”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

  NCT07265232· Lantu Biopharma· Frissítve: 2026. szept. 25.

  “Study withdrawn prior to participant enrollment following a reassessment of the clinical development plan.”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders

  NCT07254988· University of Copenhagen· Frissítve: 2026. ápr. 16.

  “Terminated since further recruitment proved difficult”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Chinese Regional Spinal Muscular Atrophy Patient Registration Study

  NCT05631418· The Children's Hospital of Zhejiang University School of Medicine· Frissítve: 2023. febr. 23.

  “Due to the lack of application materials, the normal research can not be carried out for the time being.”A regiszter szövege, változtatás nélkül. Forrás

Vizsgálatok, amelyek regiszteradata szerint az egyéni résztvevői adatokat (IPD) megosztják.

• A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

  NCT05789758· Biogen· Forrás

• AAV9-ASAH1 intracisterna magna Gene Therapy (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA - PME)

  2026-527323-10-00· Genethon· Forrás

• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

  NCT05115110· Hoffmann-La Roche· Forrás

• A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

  NCT04488133· Biogen· Forrás

• ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

  NCT07448610· Novartis Pharmaceuticals· Forrás

• A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care

  NCT04317794· Biogen· Forrás

• Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

  NCT06300996· Marco Capogrosso· Forrás

• A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

  NCT05575011· Biogen· Forrás

• A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)

  NCT07221669· Biogen· Forrás

• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

  NCT05232929· Genentech, Inc.· Forrás

Publikációk erről a betegségről egy MeSH-módszerkifejezés szerint, például szekvenálási vagy képalkotó módszerre.

• Organoids 1
• CRISPR-Cas Systems 4
• Gene Expression Profiling 1
• Whole Genome Sequencing 1
• Exome Sequencing 1
• Proteomics 4
• Metabolomics 1
• Mass Spectrometry 1
• Magnetic Resonance Imaging 13
• Machine Learning 1
• Cohort Studies 12

• Multi-Omics Landscape of Paraspinal Muscles in Spinal Muscular Atrophy With Scoliosis.

  J Cell Mol Med, 2026. júl.

• Proteomic alterations in cerebrospinal fluid of spinal muscular atrophy patients undergoing nusinersen therapy: a systematic review and meta-analysis of potential biomarkers of treatment response.

  Neurol Sci, 2026. ápr.

• Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic.

  Proc Natl Acad Sci U S A, 2026. márc.

• Longitudinal multi-omics profiling of spinal muscular atrophy.

  Neurotherapeutics, 2026. márc.

Megmutatjuk, a betegség mely célpontjait említi az absztrakt, és onnan a rajtuk dolgozókhoz jutsz.