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tag approvals 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.
Novartis’s $9.7 Billion Purchase Includes Novel PCSK9 Inhibitor
Emily Makowski | Nov 25, 2019 | 2 min read
The pharmaceutical firm is buying The Medicines Company, which recently devoted its efforts into developing the cholesterol-lowering medication.
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.
Bespoke Stem Cells for Brain Disease
Nsikan Akpan | Jan 14, 2013 | 3 min read
Scientists use virus-free gene therapy on patient-derived stem cells to repair spinal muscular atrophy in mice.
Updated July 9
Track COVID-19 Vaccines Advancing Through Clinical Trials
The Scientist | Apr 7, 2020 | 10+ min read
Find the latest updates in this one-stop resource, including efficacy data and side effects of approved shots, as well as progress on new candidates entering human studies.
2020 Top 10 Innovations
The Scientist | Dec 1, 2020 | 10+ min read
From a rapid molecular test for COVID-19 to tools that can characterize the antibodies produced in the plasma of patients recovering from the disease, this year’s winners reflect the research community’s shared focus in a challenging year.
Red blood vessels that decrease in diameter as they radiate outward are pictured on a pink and white surface
Antisense Oligonucleotides Cross Rodents’ Blood-Brain Barrier
Abby Olena, PhD | Aug 18, 2021 | 3 min read
RNA-DNA complexes that were modified with cholesterol made it into the brains of rats and mice, where they knocked down target genes.
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.

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