Understanding ALS: new therapeutic approaches

[Lymphangioleiomyomatosis: new therapeutic approaches].

Lymphangioleiomyomatosis (LAM) is a rare disease that affects young women of child-bearing age. It is characterized by the presence of cystic changes in the pulmonary parenchyma, repeated pneumothorax, chylothorax and renal angiomyolipomas, and in the majority of cases it is accompanied by a progressive loss in lung function.1 In the last two decades, there has been an increase in the understan...

Stem cells for ALS: An overview of possible therapeutic approaches.

Amyotrophic lateral sclerosis (ALS) is an unusual, fatal, neurodegenerative disorder leading to the loss of motor neurons. After diagnosis, the average lifespan ranges from 3 to 5 years, and death usually results from respiratory failure. Although the pathogenesis of ALS remains unclear, multiple factors are thought to contribute to the progression of ALS, such as network interactions between g...

The Role of TNF-Alpha in ALS: New Hypotheses for Future Therapeutic Approaches

Familial Hypercholesterolemia: new therapeutic approaches

Familial Hypercholesterolemia (FH) is caused by a mutation in the gene that encodes the low-density lipoprotein (LDL) receptor, resulting in very high levels of circulating LDL-cholesterol and endothelial damage during time. Apoprotein B, activated by LDL receptor, and PCSK9 (proprotein convertase subtilisin / kexin type 9 serine Protease), that impairs the clearance of LDL receptors, are impor...

Metabolic syndrome: new therapeutic approaches

In the last three decades in the United States the prevalence of overweight/obesity in pediatric population has more than tripled, causing the onset also in pediatric age of diseases previously considered exclusively of adults, such as metabolic syndrome (MetS) [1]. MetS represents a cluster of cardiometabolic abnormalities, including visceral obesity, dyslipidemia, hypertension and diabetes me...