by Stacey Hawkins | Dec 17, 2019 | Genetic Disease
New Muscle In The Fight Against DMD Last week’s biotech headlines lit up with the FDA’s unexpected approval of Sarepta Therapeutics’ (Cambridge, MA) newest Duchenne muscular dystrophy (DMD) drug, Vyondys 53. The FDA changed course after rejecting the product in August... 
by Stacey Hawkins | Nov 5, 2019 | Genetic Disease
A Breath of fresh air for cystic fibrosis patients It’s not often that a radical treatment for a chronic, often fatal, disease comes around. The WEEKLY takes notice when one does. Vertex Pharmaceuticals’ (Boston, MA) innovative new drug, Trikafta, promises better... 
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 | Jun 14, 2018 | Genetic Disease
Biopharma To The Rescue: PKU The ubiquitous soda can. Who hasn’t seen one? Ever look on the back, at the disturbingly long paragraph of ingredients? The list of ingredients on the back of a can of diet soda are perhaps even more unsettling. Underneath it,... 
by Stacey Hawkins | Nov 2, 2017 | Genetic Disease
You’re at the supermarket, puzzling over whether those peaches for the pie are ripe. Maybe you’re watching your child’s soccer team, and struggling to separate the Green Hornets from the Scarlet Knights. As if determining offsides isn’t hard enough! Or more seriously,... 
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...
