Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...
STAT6d
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?
Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...
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This Rare Disease Catches Infants Within First 6 Months, Could Go Unnoticed Until It’s Too Late
By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.
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Patient Recruitment for Rare Disease Trials Summit
Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...
Developing tailor-made treatments for ultra-rare disease patients: Research provides a roadmap
In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...
Science News1d
A common drug may help treat a rare genetic disease
Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...
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Arrowhead targets rare disease patients, pushing message that hope is on the horizon
Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...
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FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub
Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...
Neurogene: Interesting CNS Disease Company, Data Coming Soon
Neurogene has a $903M market cap with two phase 1/2 assets, NGN-401 for Rett Syndrome and NGN-101 for CLN5 Batten Disease.
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Rare disease advocacy is a year-round endeavor for me
While PH Awareness Month is a great opportunity to connect and educate, rare disease advocacy is columnist Jen Cueva's passion all year.
BioCryst’s Promising Prospects in Rare Disease Market Driven by BCX17725 and Orladeyo Success
In a report released today, Andrew Fein from H.C. Wainwright reiterated a Buy rating on BioCryst (BCRX – Research Report), with a price ...
Tahlequah Daily Press2d
CHIEF CHAT: Cherokee Nation rare disease summit to address overlooked health care needs
About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare Disease Summit ...