Expanding its portfolio of rare disease therapies in India, global bio-pharmaceutical company Takeda on Monday announced launch of the enzyme replacement therapy for ‘lysosomal storage disorders’ (LSD).
“Under ‘lysosomal storage disorders’, India has been recording prevalence of Hunter Syndrome, Gaucher Disease and Fabry Disease. To address these, Takeda brings to the market Idursulfase for Hunter Syndrome, Velaglucerase Alpha for Gaucher Disease and Agalsidase Alfa for Fabry Disease,” the company said in a statement.
Fabry disease interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3). Over 8,000-10,000 people worldwide have been affected by the disease.
Hunter Syndrome is a serious genetic disorder that interferes with the body’s ability to break down specific mucopolysaccharides, also known as glycosaminoglycans or GAGs. It primarily affects males.
Gaucher is a rare inherited metabolic condition that affects approximately one in 100,000 people. Patients with type-1 Gaucher disease may experience varying symptoms and degrees of disease severity, making it difficult to diagnose. (IANS)
Researchers have developed a treatment that may help reverse chemical imbalances made to the brain by habitual drug use and could one day help recovering drug addicts avoid future drug use.
When tested on rats, the new treatment was effective in reducing the animals’ cravings, according to the findings published in the Journal of Medicinal Chemistry.
When someone habitually misuses drugs, their brain chemistry is changed in ways that make it harder for them to quit taking drugs despite negative consequences.
Once someone has developed this brain disorder, their mind pays sharper attention to cues that encourage drug use, making it harder for them to abstain.
Serotonin, a brain chemical that transmits information between neural regions, is a key player in these changes.
The researchers found that the serotonin 2C receptors in drug addicts do not work as well as they should.
The team led by researchers from the University of Texas Medical Branch at Galveston in the US designed, synthesised and pharmacologically evaluated a series of small molecule therapeutics designed to restore the weakened signalling.
The findings showed that the novel therapeutic may help reverse chemical imbalances made to the brain by habitual drug use.
In their experiment, the researchers trained rats to press on a lever for cocaine infusions at certain light cues.
Once the rats learned this cocaine-seeking behaviour, half of them received the most promising therapeutic and the other half received only saline.
The findings showed that the animals treated with the new therapeutic pressed the lever for cocaine far fewer times than the saline-treated control animals, even when reinforced with the cocaine-associated light cues.
“We are the first to show that a serotonin 2C receptor therapeutic of this type can be successfully used to decrease drug-seeking behaviours,” said Kathryn Cunningham, Director of Center for Addiction Research at the University of Texas Medical Branch at Galveston.
“Our findings are especially exciting because in addition to someday helping people to recover from drug addiction, impaired functioning of the serotonin 2C receptor is also thought to contribute to other chronic health issues such as depression, impulsivity disorders, obesity and schizophrenia,” Cunningham added. (IANS)