Hope On The Horizon For Huntington’s

Hope On The Horizon For Huntington’s

Hope on the horizon for HUNTINGTON’S No question about it, Huntington’s disease (HD) is cruel. In the beginning, sufferers of this fatal neurodegenerative disorder experience involuntary movement and mood disturbance, most often in the form of depression. As the...
Exon-Skipping Therapy For DMD

Exon-Skipping Therapy For DMD

The Science Behind Sarepta’s Hotly Debated Antisense Drug Sarepta’s (Cambridge, MA) Duchenne muscular dystrophy drug Exondys 51 crossed the finish line earlier this week, with a conditional stamp of approval by the FDA. This hotly debated regulatory result...
Drugging The Undruggable

Drugging The Undruggable

Antagonists Fight A Good Fight Small molecule inhibitors, also known as antagonists in the industry, fight a good fight. In fact, many drugs on the market today work by inhibiting overactive, disease-associated proteins. Novartis’ (Basel, Switzerland) top...
Antisense, RNAi And MicroRNA Explained

Antisense, RNAi And MicroRNA Explained

Make Way For RNA Based Therapies The up hill battle of RNA therapeutics to the clinic continues despite extensive use in research. Recall from high school biology that RNA translates DNA code into a language ribosomes can understand in order to make proteins required...