New drug slows Niemann–Pick disease

Parkinson's disease--challenges in new drug development.

Idiopathic Parkinson's disease (IPD) is the second most common neurodegenerative disorder after Alzheimer's disease. Treatment aims in IPD include the provision of symptomatic relief reduction of functional disability, halting or slowing of the neurodegenerative process, and the prevention of long-term complications by proper initiation of therapy. At present, pharmacotherapeutic strategies all...

Inhibition of Activin Signaling Slows Progression of Polycystic Kidney Disease.

Autosomal dominant polycystic kidney disease (ADPKD), characterized by the formation of numerous kidney cysts, is caused by PKD1 or PKD2 mutations and affects 0.1% of the population. Although recent clinical studies indicate that reduction of cAMP levels slows progression of PKD, this finding has not led to an established safe and effective therapy for patients, indicating the need to find new ...

2-Hydroxyestradiol slows progression of experimental polycystic kidney disease.

Male gender is a risk factor for progression of polycystic kidney disease (PKD). 17β-Estradiol (E2) protects experimentally, but clinical use is limited by adverse effects. Novel E2 metabolites provide many benefits of E2 without stimulating the estrogen receptor, and thus may be safer. We hypothesized that E2 metabolites are protective in a model of PKD. Studies were performed in male control ...

Folate-conjugated rapamycin slows progression of polycystic kidney disease.

Activation of the mammalian target of rapamycin (mTOR) signaling pathway is aberrant in autosomal-dominant polycystic kidney disease (ADPKD). The mTOR inhibitors, such as rapamycin, ameliorate PKD in rodent models, but clinical trials have not shown benefit, possibly as a result of low tissue concentrations of rapamycin at clinically tolerable doses. To overcome this limitation, we synthesized ...

New Dimension in Drug Addiction Therapy