Friedreich-ataxia: ki dolgozik rajta

Intézményenként egy kártya. Célpontra vagy országra szűkítheted a listát.

• Minden célpont
• NRF2 0
• BRD4 0
• KIT 0
• NTRK 0
• PPAR 0
• RUNX1 0
• STING 0

84 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.

• NIH NINDS · F31

  Determining a Mechanism for TGF-beta-activated kinase 1 (TAK1) Regulation of Nemo-like kinase (NLK) in Spinocerebellar Ataxia Type 1 (SCA1)

  Yale University· New Haven, Egyesült Államok· 50 114 USD awarded in fiscal year 2026· until 2029. szept.

• NIH NIAID · R56

  Functional dissection of a novel E2 complex critical for the HPV16 life cycle

  Virginia Commonwealth University· Richmond, Egyesült Államok· 768 308 USD awarded in fiscal year 2026· until 2027. aug.

• NIH NINDS · R61

  CRISPR/Cas9-Mediated Therapeutic Targeting of CAG Repeats in Spinocerebellar Ataxia Type 12

  Johns Hopkins University· Baltimore, Egyesült Államok· 581 250 USD awarded in fiscal year 2026· until 2028. febr.

• NIH NINDS · R56

  The Mechanism of Trinucleotide Repeat Disease Expansions

  Ohio State University· Columbus, Egyesült Államok· 551 250 USD awarded in fiscal year 2026· until 2027. aug.

• NIH NINDS · RM1

  DECIPHERING THE MOLECULAR BASIS OF SOMATIC GAA-REPEAT DYNAMICS IN SCA27B

  University of Miami School of Medicine· Coral Gables, Egyesült Államok· 1 819 495 USD awarded in fiscal year 2026· until 2031. júl.

• European Commission (Horizon Europe) · HORIZON-ERC

  Engineering Synthetic Gene Circuits through Competitive Dimerization. (DIMERCIRCUITS)

  Fondazione Telethon Ets· Roma, Olaszország· EU contribution 2 484 115 EUR· until 2030. dec.

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

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

  Discrete topological and conformal quantum field theory through combinatorial factorisation algebras (DisQ FA)

  Aalto Korkeakoulusaatio Sr· Espoo, Finnország· EU contribution 226 277 EUR· until 2027. aug.· 2 countries

• European Commission (Horizon Europe) · HORIZON-ERC

  Genomic observation and perturbation: genetic modifiers to untangle disease mechanisms of RFC1 repeat expansion (GEMREX)

  University College London· London, Egyesült Királyság· EU contribution 1 500 000 EUR· until 2030. ápr.

• European Commission (Horizon Europe) · HORIZON-RIA

  PHOENIX: Precision Human Organs-on-chip for Enhanced Neuro-muscular and Cardiac Studies

  Politecnico di Milano· Milano, Olaszország· EU contribution 7 095 163 EUR· until 2028. dec.· 5 countries

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

• European Commission (Horizon Europe) · HORIZON-ERC

  Illuminating human Fe-S cluster biology (FeSurveil)

  Weizmann Institute of Science· Rehovot, Izrael· EU contribution 1 499 999 EUR· until 2029. aug.

10 of 343 running projects, newest first. Listed amounts together: 177,4 M USD + 18,2 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.

• Leállították

  A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

  NCT06681766· Larimar Therapeutics, Inc.· Frissítve: 2026. júl. 10.

  “Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

  NCT06483802· Astellas Gene Therapies· Frissítve: 2025. nov. 28.

  “Corporate decision to withdraw the trial at this time.”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

  NCT06560346· Friedreich's Ataxia Research Alliance· Frissítve: 2025. jún. 8.

  “Lack of Funding”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

  NCT03761511· RWTH Aachen University· Frissítve: 2024. jan. 24.

  “Due to a temporary halt the recruitment start was delayed. A re-evaluation of the sponsor revealed, that the study is no longer feasible due to increased administrative and financial requirements.”A regiszter szövege, változtatás nélkül. Forrás

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

• Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.

  NCT07095062· IRCCS Eugenio Medea· Forrás

• Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia

  NCT06692296· IRCCS Eugenio Medea· Forrás

• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

  NCT01793168· Sanford Health· Forrás

• A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World

  NCT07887828· Biogen· Forrás

• A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

  NCT06953583· Biogen· Forrás

• Cardiac Output and Fatigue in Friedreich's Ataxia

  NCT07444333· Scott Barbuto· Forrás

• A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

  NCT02255435· Biogen· Forrás

• A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

  NCT06054893· Biogen· Forrás

• A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

  NCT06628687· Biogen· Forrás

• A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

  NCT06623890· Biogen· Forrás

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

• Organoids 2
• CRISPR-Cas Systems 3
• Gene Expression Profiling 1
• Whole Genome Sequencing 1
• Exome Sequencing 1
• Immunohistochemistry 1
• Magnetic Resonance Imaging 5
• Machine Learning 1
• Cohort Studies 1

• Generation of Spontaneously Beating Neuromyocardial Organoids with Neural Crest-Derived Proprioceptive-Biased Neurons and Real-Time Respirometry.

  J Vis Exp, 2026. okt.

• Modeling Friedreich's ataxia with Bergmann glia-enriched human cerebellar organoids.

  Commun Biol, 2026. máj.

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