deferiprone has been researched along with Friedreich Ataxia in 15 studies
Deferiprone: A pyridone derivative and iron chelator that is used in the treatment of IRON OVERLOAD in patients with THALASSEMIA.
deferiprone : A member of the class of 4-pyridones that is pyridin-4(1H)-one substituted at positions 1 and 2 by methyl groups and at position 3 by a hydroxy group. A lipid-soluble iron-chelator used for treatment of thalassaemia.
Friedreich Ataxia: An autosomal recessive disease, usually of childhood onset, characterized pathologically by degeneration of the spinocerebellar tracts, posterior columns, and to a lesser extent the corticospinal tracts. Clinical manifestations include GAIT ATAXIA, pes cavus, speech impairment, lateral curvature of spine, rhythmic head tremor, kyphoscoliosis, congestive heart failure (secondary to a cardiomyopathy), and lower extremity weakness. Most forms of this condition are associated with a mutation in a gene on chromosome 9, at band q13, which codes for the mitochondrial protein frataxin. (From Adams et al., Principles of Neurology, 6th ed, p1081; N Engl J Med 1996 Oct 17;335(16):1169-75) The severity of Friedreich ataxia associated with expansion of GAA repeats in the first intron of the frataxin gene correlates with the number of trinucleotide repeats. (From Durr et al, N Engl J Med 1996 Oct 17;335(16):1169-75)
| Excerpt | Relevance | Reference |
|---|---|---|
| "We conducted a 6-month, randomized, double-blind, placebo-controlled study to assess safety, tolerability, and efficacy of deferiprone in Friedreich ataxia (FRDA)." | 9.19 | Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. ( Arpa, J; Delatycki, MB; Le Quan Sang, KH; Mariotti, C; Munnich, A; Pandolfo, M; Sanz-Gallego, I; Spino, M; Tai, G; Tarnopolsky, MA; Taroni, F; Tricta, F, 2014) |
| "Friedreich ataxia is an inherited disorder characterized by degeneration of the peripheral and central nervous system and hypertrophic cardiomyopathy." | 6.82 | Clinical Experience With Deferiprone Treatment for Friedreich Ataxia. ( Arad, M; Cabantchik, I; Elincx-Benizri, S; Freimark, D; Glik, A; Hassin-Baer, S; Kozlova, E; Merkel, D, 2016) |
| "We conducted a 6-month, randomized, double-blind, placebo-controlled study to assess safety, tolerability, and efficacy of deferiprone in Friedreich ataxia (FRDA)." | 5.19 | Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. ( Arpa, J; Delatycki, MB; Le Quan Sang, KH; Mariotti, C; Munnich, A; Pandolfo, M; Sanz-Gallego, I; Spino, M; Tai, G; Tarnopolsky, MA; Taroni, F; Tricta, F, 2014) |
| " Despite that many chelators inhibit FRD in vitro and in vivo, only Deferiprone (L1) has been shown to be effective and safe in the reversal of oxidative stress related tissue damage in iron overload and other conditions such as cardiomyopathy, acute kidney disease, Friedreich ataxia etc." | 4.90 | Antioxidant targeting by deferiprone in diseases related to oxidative damage. ( Kolnagou, A; Kontoghiorghe, CN; Kontoghiorghes, GJ, 2014) |
| "Friedreich ataxia is an inherited disorder characterized by degeneration of the peripheral and central nervous system and hypertrophic cardiomyopathy." | 2.82 | Clinical Experience With Deferiprone Treatment for Friedreich Ataxia. ( Arad, M; Cabantchik, I; Elincx-Benizri, S; Freimark, D; Glik, A; Hassin-Baer, S; Kozlova, E; Merkel, D, 2016) |
| "Friedreich's Ataxia is an autosomal recessive genetic disease causing the defective gene product, frataxin." | 2.72 | Friedreich Ataxia: current state-of-the-art, and future prospects for mitochondrial-focused therapies. ( Gonzalez-Cabo, P; Lyakhovich, A; Pagano, G; Pallardó, FV; Rodríguez, LR; Trifuoggi, M, 2021) |
| "It is generally accepted that Friedreich's ataxia (FRDA) is caused by a deficiency in frataxin expression, a mitochondrial protein involved in iron homeostasis, which mainly affects the brain, dorsal root ganglia of the spinal cord, heart and in certain cases the pancreas." | 1.42 | Novel aberrant genetic and epigenetic events in Friedreich's ataxia. ( Jones, J; Martinez, S; Moraleda, JM; Quesada, MP; Rodríguez-Lozano, FJ, 2015) |
| "Twenty Friedreich's ataxia patients were treated with idebenone (20 mg/kg/day) and deferiprone (20 mg/kg/day) for 11 months." | 1.37 | Combined therapy with idebenone and deferiprone in patients with Friedreich's ataxia. ( Aracil, A; Artuch, R; Blanch, J; Capdevila, A; Jiménez, L; Mas, A; Montero, R; O'Callaghan, M; Pineda, M; Tondo, M; Velasco-Sánchez, D, 2011) |
| "Friedreich ataxia is a neurological disease originating from an iron-sulfur cluster enzyme deficiency due to impaired iron handling in the mitochondrion, aconitase being particularly affected." | 1.35 | Deferiprone targets aconitase: implication for Friedreich's ataxia treatment. ( Dassa, EP; Goncalves, S; Paupe, V; Rustin, P, 2008) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 4 (26.67) | 29.6817 |
| 2010's | 9 (60.00) | 24.3611 |
| 2020's | 2 (13.33) | 2.80 |
| Authors | Studies |
|---|---|
| Börklü, E | 1 |
| Pallardó, FV | 1 |
| Pagano, G | 1 |
| Rodríguez, LR | 1 |
| Gonzalez-Cabo, P | 1 |
| Lyakhovich, A | 1 |
| Trifuoggi, M | 1 |
| Soriano, S | 1 |
| Llorens, JV | 1 |
| Blanco-Sobero, L | 1 |
| Gutiérrez, L | 1 |
| Calap-Quintana, P | 1 |
| Morales, MP | 1 |
| Moltó, MD | 1 |
| Martínez-Sebastián, MJ | 1 |
| Arpa, J | 2 |
| Sanz-Gallego, I | 2 |
| Rodríguez-de-Rivera, FJ | 1 |
| Domínguez-Melcón, FJ | 1 |
| Prefasi, D | 1 |
| Oliva-Navarro, J | 1 |
| Moreno-Yangüela, M | 1 |
| Pandolfo, M | 2 |
| Hausmann, L | 1 |
| Kontoghiorghe, CN | 1 |
| Kolnagou, A | 1 |
| Kontoghiorghes, GJ | 1 |
| Delatycki, MB | 1 |
| Le Quan Sang, KH | 2 |
| Mariotti, C | 1 |
| Munnich, A | 3 |
| Tai, G | 1 |
| Tarnopolsky, MA | 1 |
| Taroni, F | 1 |
| Spino, M | 1 |
| Tricta, F | 1 |
| Quesada, MP | 1 |
| Jones, J | 1 |
| Rodríguez-Lozano, FJ | 1 |
| Moraleda, JM | 1 |
| Martinez, S | 1 |
| Lee, YK | 1 |
| Lau, YM | 1 |
| Ng, KM | 1 |
| Lai, WH | 1 |
| Ho, SL | 1 |
| Tse, HF | 1 |
| Siu, CW | 1 |
| Ho, PW | 1 |
| Elincx-Benizri, S | 1 |
| Glik, A | 1 |
| Merkel, D | 1 |
| Arad, M | 1 |
| Freimark, D | 1 |
| Kozlova, E | 1 |
| Cabantchik, I | 1 |
| Hassin-Baer, S | 1 |
| Goncalves, S | 1 |
| Paupe, V | 1 |
| Dassa, EP | 1 |
| Rustin, P | 1 |
| Kakhlon, O | 1 |
| Manning, H | 1 |
| Breuer, W | 1 |
| Melamed-Book, N | 1 |
| Lu, C | 1 |
| Cortopassi, G | 1 |
| Cabantchik, ZI | 2 |
| Tsou, AY | 1 |
| Friedman, LS | 1 |
| Wilson, RB | 1 |
| Lynch, DR | 1 |
| Velasco-Sánchez, D | 1 |
| Aracil, A | 1 |
| Montero, R | 1 |
| Mas, A | 1 |
| Jiménez, L | 1 |
| O'Callaghan, M | 1 |
| Tondo, M | 1 |
| Capdevila, A | 1 |
| Blanch, J | 1 |
| Artuch, R | 1 |
| Pineda, M | 1 |
| Boddaert, N | 1 |
| Rötig, A | 1 |
| Leroy-Willig, A | 1 |
| Gallet, S | 1 |
| Brunelle, F | 1 |
| Sidi, D | 1 |
| Thalabard, JC | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy[NCT02882477] | Phase 2/Phase 3 | 20 participants (Anticipated) | Interventional | 2016-12-31 | Not yet recruiting | ||
| Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis[NCT02164253] | Phase 2 | 23 participants (Actual) | Interventional | 2013-09-30 | Completed | ||
| A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease[NCT01539837] | Phase 2 | 22 participants (Actual) | Interventional | 2012-02-29 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
Assess whether Deferiprone therapy directly affects the symptoms of Parkinson's disease, modify regional brain mineralization (iron concentration) as assessed with T2* MRI in PD patients in the dentate nucleus. In previous animal studies, Deferiprone treatment reduced dentate nucleus iron content, as assessed by MRI. An increase in the T2*MRI value represents an increase in mineralization. (NCT01539837)
Timeframe: 6 months
| Intervention | ms (Mean) |
|---|---|
| Placebo | 30.74 |
| Deferiprone 20mg | 30.59 |
| Deferiprone 30mg | 29.86 |
To assess whether there were any serious adverse events in 6-month treatment with Deferiprone. (NCT01539837)
Timeframe: 6 months
| Intervention | Participants (Count of Participants) |
|---|---|
| Placebo | 0 |
| Deferiprone 20mg | 0 |
| Deferiprone 30mg | 0 |
4 reviews available for deferiprone and Friedreich Ataxia
| Article | Year |
|---|---|
Friedreich Ataxia: current state-of-the-art, and future prospects for mitochondrial-focused therapies.
Topics: Animals; Antioxidants; Carnitine; Deferiprone; Friedreich Ataxia; Humans; Iron Chelating Agents; Lin | 2021 |
Deferiprone for the treatment of Friedreich's ataxia.
Topics: Animals; Clinical Trials as Topic; Deferiprone; Drug Evaluation, Preclinical; Frataxin; Friedreich A | 2013 |
Antioxidant targeting by deferiprone in diseases related to oxidative damage.
Topics: Acute Kidney Injury; Antioxidants; Cardiomyopathies; Deferiprone; Free Radicals; Friedreich Ataxia; | 2014 |
Pharmacotherapy for Friedreich ataxia.
Topics: Animals; Antioxidants; Deferiprone; Enzyme Inhibitors; Erythropoietin; Friedreich Ataxia; Histone De | 2009 |
4 trials available for deferiprone and Friedreich Ataxia
| Article | Year |
|---|---|
Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia - open-label trial.
Topics: Adolescent; Adult; Deferiprone; Female; Friedreich Ataxia; Humans; Male; Middle Aged; Pilot Projects | 2014 |
Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial.
Topics: Adolescent; Adult; Child; Deferiprone; Disability Evaluation; Dose-Response Relationship, Drug; Doub | 2014 |
Clinical Experience With Deferiprone Treatment for Friedreich Ataxia.
Topics: Adolescent; Adult; Antioxidants; Deferiprone; Disease Progression; Double-Blind Method; Drug Therapy | 2016 |
Selective iron chelation in Friedreich ataxia: biologic and clinical implications.
Topics: Adolescent; Adult; Biological Transport; Brain; Brain Chemistry; Case-Control Studies; Deferiprone; | 2007 |
Selective iron chelation in Friedreich ataxia: biologic and clinical implications.
Topics: Adolescent; Adult; Biological Transport; Brain; Brain Chemistry; Case-Control Studies; Deferiprone; | 2007 |
Selective iron chelation in Friedreich ataxia: biologic and clinical implications.
Topics: Adolescent; Adult; Biological Transport; Brain; Brain Chemistry; Case-Control Studies; Deferiprone; | 2007 |
Selective iron chelation in Friedreich ataxia: biologic and clinical implications.
Topics: Adolescent; Adult; Biological Transport; Brain; Brain Chemistry; Case-Control Studies; Deferiprone; | 2007 |
7 other studies available for deferiprone and Friedreich Ataxia
| Article | Year |
|---|---|
Insights from yeast: Transcriptional reprogramming following metformin treatment is similar to that of deferiprone in a yeast Friedreich's ataxia model.
Topics: Deferiprone; Diabetes Mellitus, Type 2; Friedreich Ataxia; Humans; Iron; Metformin; Saccharomyces ce | 2023 |
Deferiprone and idebenone rescue frataxin depletion phenotypes in a Drosophila model of Friedreich's ataxia.
Topics: Aconitate Hydratase; Animals; Antioxidants; Deferiprone; Disease Models, Animal; Drosophila; Frataxi | 2013 |
Novel aberrant genetic and epigenetic events in Friedreich's ataxia.
Topics: Antioxidants; Apoptosis; Azacitidine; Brain-Derived Neurotrophic Factor; Caspase 3; Cells, Cultured; | 2015 |
Efficient attenuation of Friedreich's ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA.
Topics: Antioxidants; Deferiprone; Drug Evaluation, Preclinical; Frataxin; Friedreich Ataxia; Gene Expressio | 2016 |
Deferiprone targets aconitase: implication for Friedreich's ataxia treatment.
Topics: Aconitate Hydratase; Cell Proliferation; Cells, Cultured; Cytosol; Deferiprone; Fibroblasts; Friedre | 2008 |
Cell functions impaired by frataxin deficiency are restored by drug-mediated iron relocation.
Topics: Adenosine Triphosphate; Cell Line; Deferiprone; DNA Damage; DNA, Mitochondrial; Frataxin; Friedreich | 2008 |
Combined therapy with idebenone and deferiprone in patients with Friedreich's ataxia.
Topics: Adolescent; Adult; Antioxidants; Blood Cell Count; Brain Chemistry; Child; Deferiprone; Drug Therapy | 2011 |