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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.

Treatment 133
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The Long Road to End Tuberculosis

Codon

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.

Vaccine 114
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FDA clears Nuvation lung cancer drug, setting up battle with Bristol Myers and Roche

BioPharma Drive: Drug Pricing

Ibtrozi has also shown the ability to help people whose cancer has spread to the brain, a leading cause of disease progression, the company said. In a research note on Wednesday, RBC Capital Markets analyst Leonid Timashev estimated peak yearly sales of the drug could reach nearly $640 million.

FDA 269
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Edwin Cohn and the Harvard Blood Factory

Codon

The same team also invented several other products that proved useful for treating battlefield injuries, including gamma globulins to inoculate soldiers against diseases and fibrin film to minimize bleeding during brain surgeries. In 1929 and 1930, two medical doctors working with Cohn — George R. Minot and William P.

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Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

BioPharma Drive: Drug Pricing

On a conference call held Wednesday afternoon, Sarepta CEO Doug Ingram said that "failure to adapt" to recent setbacks "would risk our long-term viability as an organization and decrease the opportunity to bring the greatest benefit to the greatest number of patients living with rare disease." Please select at least one newsletter.

Therapies 272
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Moderna COVID vaccine gets full approval for children

BioPharma Drive: Drug Pricing

Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Moderna COVID vaccine gets full approval for children The approval comes amid regulatory upheaval under HHS head Robert F. Kennedy Jr., Kennedy Jr.

Vaccine 226
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Soleno sales of new Prader-Willi drug rise faster than expected

BioPharma Drive: Drug Pricing

Spencer Platt via Getty Images The first marketed treatment for the insatiable hunger associated with Prader-Willi disease is selling more quickly than Wall Street analysts expected, an early, but encouraging sign of demand. The disease, which affects an estimated 10,000 to 20,000 people in the U.S.,

Drugs 215