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Pharma Looks to Inflammasome Inhibitors as All-Around Therapies
Pharma Looks to Inflammasome Inhibitors as All-Around Therapies
Many major biopharmaceutical companies are developing or acquiring drugs that target the NLRP3 inflammasome, a large intracellular complex that researchers say can spark inflammation and stoke diseases of lifestyle and aging.
Pharma Looks to Inflammasome Inhibitors as All-Around Therapies
Pharma Looks to Inflammasome Inhibitors as All-Around Therapies

Many major biopharmaceutical companies are developing or acquiring drugs that target the NLRP3 inflammasome, a large intracellular complex that researchers say can spark inflammation and stoke diseases of lifestyle and aging.

Many major biopharmaceutical companies are developing or acquiring drugs that target the NLRP3 inflammasome, a large intracellular complex that researchers say can spark inflammation and stoke diseases of lifestyle and aging.

rare diseases, disease & medicine

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Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez, PhD | Dec 11, 2019 | 4 min read
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
AI App Identifies Rare Genetic Disorders from Photos of Patients’ Faces
Catherine Offord | Jan 8, 2019 | 2 min read
Deep-learning algorithms could help doctors narrow in on the causes of certain medical conditions, say researchers.
First RNAi Therapy Approved by FDA
Kerry Grens | Aug 10, 2018 | 1 min read
Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018 | 2 min read
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
In Utero Transplant in First Clinical Trial Successful
Jim Daley | May 30, 2018 | 2 min read
Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.
New Treatments for Phenylketonuria Aim to Loosen Reins on Strict Diet
Diana Kwon | May 29, 2018 | 5 min read
Biotechs have developed enzyme replacements and genetically modified probiotics to treat patients with the rare metabolic disorder.
Patient Registries to Bolster Cell- and Gene-Therapy Clinical Trial Data
Anna Azvolinsky | May 15, 2018 | 3 min read
As the first personalized cell and gene therapies are approved from small clinical trials, researchers propose the creation of publicly accessible databases to pull together real-world results.  
Bullets and Ballots
Bob Grant | May 1, 2018 | 2 min read
Researching the rare, but all-too-common, disease of gun violence in America will take a concerted political effort.
Birth Defects Linked to Increased Risk of Childhood Cancer
Jim Daley | Apr 15, 2018 | 2 min read
Certain non-chromosomal defects are strongly associated with specific childhood cancers.
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