by Stacey Hawkins | Apr 12, 2020 | Neuroscience
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... 
by Stacey Hawkins | Jan 10, 2019 | Genetic Disease
Stump The Clumps: New Drugs To Treat Amyloidosis We at the WEEKLY aim to keep you up on the coolest, latest innovations in biotech. Bonus: we might also help you win Jeopardy. Ready? The category is Rare Diseases for $1,000. The clue is “Amyloidosis.” Ding ding ding!... 
by Stacey Hawkins | Apr 27, 2017 | RNA Therapeutics
Taking Steps With Antisense With their high specificity and relative low manufacturing cost, RNA therapeutics may be tomorrow’s biotech sweetheart. In fact, chances are good that previously “undruggable” targets that cannot be accessed by small or large molecule... 
by Stacey Hawkins | Feb 9, 2017 | Genetic Disease
FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy... 
by Stacey Hawkins | Sep 22, 2016 | RNA Therapeutics
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...
