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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 disease progresses, patients lose the...
The Infinitesimal Engines of Biologics
Although all drugs restore or improve our health, the methods used to manufacture them vary greatly. Small molecule drugs are chemically synthesized; the production of biologics revolves around cells. Pharmaceutical scientists “program” cells to make proteins by...
Curing the Leading Hereditary Cause of Infant Death
To help bring awareness to Spinal Muscular Atrophy (SMA), the leading hereditary cause of infant death, August has been named National Spinal Muscular Atrophy (SMA) month. In this Primer, we’ll explain SMA and the different therapeutic approaches to tackle this...
Alzheimer’s Disease: The Way Forward
Previously, we reviewed the pathology and genetics of Alzheimer’s disease (AD) and discussed the two most common AD drug development targets, amyloid-beta plaques, and tau. Now, we’ll look at various novel AD drug targets and the companies forging ahead to find new...
New Gene Therapy Drug Explained
In March 2023, Novartis’ (Basel, Switzerland), shared its latest data concerning Zolgensma. The findings reveal sustained durability up to 7.5 years post-dosing, with a 100% achievement of all assessed milestones in children treated before the onset of spinal muscular...
RNA Therapeutics March Onward
With their high specificity and relatively low manufacturing cost, RNA therapeutics may be tomorrow’s biotech sweetheart. In fact, chances are good that previously “undruggable” targets that small or large-molecule drugs cannot access are now within reach. However,...
Fountains of Healthy Old Age?
Many famous people have uttered (or supposedly uttered) lots of pithy comments about aging. A couple of gems: “Age is mind over matter; if you don’t mind, it don’t matter,” said Satchel Paige. “We are always the same age inside,” Gertrude Stein wrote — she lived in...
Gluten: Protein of Doom
Imagine the tantalizing scent of freshly baked bread. Now, think about even a single scrumptious slice wreaking havoc on you: stomach pain, bloating, and other digestive consequences. Welcome to Planet Celiac— home to the millions of Americans diagnosed with celiac...
Ticked Off: the Science Behind Lyme Disease
Alleluia! Spring! It means the promise of more time in the fresh air – gardening, camping, or just enjoying the sunshine. Unfortunately, time outdoors increases the risk of contracting Lyme disease. As you may know, this illness comes courtesy of our favorite...
From Drug Discovery to Approval: Phase IV
We previously focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of truth: does the study data support...
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