Fanconi syndrome future or investigational therapies

Jump to navigation Jump to search

Some of the recently introduced strategies in the management of Fanconi syndrome are provided below; of note, due to various underlying mechanisms leading to the disease, researches are on in this field[1].

  • In a rare variant of Fanconi syndrome named Fanconi reno-tubular syndrome 1 (FRTS1), the patients have fatty acid oxidation problem due to a mitochondrial defect; dequalinium chloride (DECA) which s a newly introduced drug for hyperoxaluria has appeared to be effective in treatment of this syndrome by not permitting the import of unfunctional mutated protein.
  • In other types of mitochondrial defects leading to Fanconi syndrome, it is of recently proposed that enhancement of this protein import by the drug sodium pyrithione can alleviate the disease.
  • Consumption of different anti-oxidants has shown promising results in the treatment of Fanconi syndrome with fatty acid oxidation defects.
  • It has been shown that Anti-apoptotic drugs are also very effective in Fanconi syndrome variants with cell apoptosis as a leading mechanism like tyrosinemia and cystinosis.
  • Stimulation of mammalian target of rapamycin complex 1 (mTORC1), an important regulator protein in cell autophagy and lipid metabolism, by specific aminoacids or kinases is also recently suggested as a therapeutic approach for Fanconi syndrome.
  • RNA silencing therapies are just recently introduced treatments targeting the down-regulation of disease genes with dominant inheritance and for instance the regulator microRNA mir21 is proposed to be investigated as a therapeutic target for some variants of Fanconi syndrome.

Fanconi syndrome Microchapters

Home

Patient Information

Overview

Historical Perspective

Classification

Pathophysiology

Causes

Differentiating Fanconi syndrome from other Diseases

Epidemiology and Demographics

Risk Factors

Screening

Natural History, Complications and Prognosis

Diagnosis

Diagnostic Study Of Choice

History and Symptoms

Physical Examination

Laboratory Findings

X Ray

CT scan

MRI

Other Imaging Findings

Other Diagnostic Studies

Treatment

Medical Therapy

Surgery

Primary Prevention

Secondary Prevention

Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

Fanconi syndrome future or investigational therapies On the Web

Most recent articles

Most cited articles

Review articles

CME Programs

Powerpoint slides

Images

American Roentgen Ray Society Images of Fanconi syndrome future or investigational therapies

All Images
X-rays
Echo & Ultrasound
CT Images
MRI

Ongoing Trials at Clinical Trials.gov

US National Guidelines Clearinghouse

NICE Guidance

FDA on Fanconi syndrome future or investigational therapies

CDC on Fanconi syndrome future or investigational therapies

Fanconi syndrome future or investigational therapies in the news

Blogs on Fanconi syndrome future or investigational therapies

Directions to Hospitals Treating Fanconi syndrome

Risk calculators and risk factors for Fanconi syndrome future or investigational therapies

References

  1. Krasnova TN, Samokhodskaya LM, Ivanitsky LV, Korogodina AD, Borisov EN, Nikiforova NV; et al. (2015). "[Impact of interleukin-10 and interleukin-28 gene polymorphisms on the development and course of lupus nephritis]". Ter Arkh. 87 (6): 40–44. doi:10.17116/terarkh201587640-44. PMID 26281194.