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tag fda disease medicine

FDA Approves Oral Drug for Fabry Disease
Ashley Yeager | Aug 13, 2018 | 2 min read
The medicine increases the activity of a deficient enzyme in certain patients with the condition.
a photo of the packaging for the drug Trikafta
FDA Approves New Cystic Fibrosis Drug
Shawna Williams | Oct 24, 2019 | 1 min read
The treatment, Trikafta, increases lung function in most patients with the disease—but comes with a hefty price tag.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019 | 3 min read
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
2022 Top 10 Innovations 
2022 Top 10 Innovations
The Scientist | Dec 12, 2022 | 10+ min read
This year’s crop of winning products features many with a clinical focus and others that represent significant advances in sequencing, single-cell analysis, and more.
Concerns over Efficacy and Cost of Muscle Wasting Treatments
Ruth Williams | Nov 11, 2020 | 5 min read
Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.
Updated Mar 8
A healthcare worker holds up three syringes with clear medicine
To Booster or Not: Scientists and Regulators Debate
Jef Akst | Sep 16, 2021 | 7 min read
President Biden’s planned rollout of additional COVID-19 vaccine doses is set to begin next week, but questions remain about who should get them.
Top 10 Innovations 2021
2021 Top 10 Innovations
The Scientist | Dec 1, 2021 | 10+ min read
The COVID-19 pandemic is still with us. Biomedical innovation has rallied to address that pressing concern while continuing to tackle broader research challenges.
Antibody Alternatives
Paul Ko Ferrigno and Jane McLeod | Feb 1, 2016 | 10+ min read
Nucleic acid aptamers and protein scaffolds could change the way researchers study biological processes and treat disease.
The Proteasome: A Powerful Target for Manipulating Protein Levels
John Hines and Craig M. Crews | May 1, 2017 | 10+ min read
The proteasome’s ability to target and degrade specific proteins is proving useful to researchers studying protein function or developing treatments for diseases.
Cannabinoid controversy
Tia Ghose | Sep 9, 2009 | 5 min read
Receptors that bind the active ingredient in marijuana may be novel therapeutic targets in autoimmune disease, but contested evidence for their presence on neurons could hamper drug development

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