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- Indexed articles, last 90 days
- 6
- Latest publication
- Aug 28, 2026
- Outlet visibility, for CNBC
- Top 5K sites
- Earliest in this view
- Jul 31, 2026
Latest articles
BioCryst is profitable. Now it wants to buy more rare disease drugs (opens the original)
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases. The science behind rare disease drug development is changing fast, and with it, so is the financial engine that drives it all. This month saw the launch of Rare Ventures â a new venture philanthropy-based accelerator lead by EB Research Partnership CEO, Michael Hund. The launch comes thanks to an
BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment (opens the original)
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases. BioMarin Pharmaceutical is making more waves on the M&A front. The company announced Tuesday that it will acquire Alesta Therapeutics for $275 million upfront. The deal gives BioMarin access to Alesta's oral treatment for hypophosphatasia, or HPP â a rare genetic bone disease. The drug, ALE1, is cur
The startup using AI to help rare disease families develop custom treatments (opens the original)
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases. Jacalyn Lee first sensed something was off with her daughter, Isla, just before her first birthday. "She was just under a year old, and nobody believed me, including the pediatrician," she said. Isla was missing some of the developmental milestones that her sisters had hit at her age. Doctors told Lee
The power of genetic testing: new screening methods are catching rare diseases before symptoms start (opens the original)
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases. For more stories like this, sign up for the CNBC Cures newsletter. In an academic article published in June, a team of researchers from New York and Boston highlighted the case of a 6-month old girl in New York diagnosed with Batten disease, a rare, fatal genetic condition that impacts the nervous
CAMP4 advances rare disease treatment for SYNGAP1 into human trials (opens the original)
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases. For more stories like this, sign up for the CNBC Cures newsletter. Regulators in Australia are providing hope to thousands of patients around the world that suffer from a rare genetic disease that currently has no approved treatment. Late last month, CAMP4 Therapeutics was given permission by Austral
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