Tag: rare disease

Market News

BioCryst Profitable Growth Sparks Rare Disease Drug Acquisitions

BioCryst just crossed into steady profits and suddenly has cash to spare. Instead of another internal gamble, the company is...

28/08/2026

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Market News

BioMarin Buys Alesta For Rare Bone Disease Drug

BioMarin just dropped another major rare-disease move, paying $275 million upfront for a promising oral treatment that could change life...

19/08/2026

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Market News

New Medicaid Work Rules Threaten Coverage for Rare Disease Patients

New Medicaid work rules set to take effect soon could disrupt coverage for thousands of rare disease patients who struggle...

31/07/2026

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Market News

Trump Ousts FDA Commissioner Marty Makary After Backlash

When President Trump announced Marty Makary was out as FDA Commissioner, it marked the end of a turbulent chapter filled...

12/05/2026

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Regeneron Gene Therapy for Rare Hearing Loss Goes Free in US

Regeneron just announced its new gene therapy for a rare form of genetic hearing loss will be completely free in...

25/04/2026

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From Paralysis to Boston Marathon: One Woman’s Fight Against Rare Disease

Imagine waking up unable to walk after a mysterious illness strikes, doctors unsure if you'd ever move freely again. One...

20/04/2026

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Mom’s AI Advocate Transforms Rare Disease Care for Families

When a mom faced her daughter's ultra-rare diagnosis with almost no guidance, she didn't just advocate — she built an...

11/04/2026

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Raising a Child With Angelman Syndrome: A Doctor’s Personal Journey

When a doctor receives his own son's rare disease diagnosis, everything changes. What started as grief in a hospital parking...

10/04/2026

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Breakthrough Hope: New Treatment for Rare KIF1A Gene Disorder

When a toddler stopped kicking in the bath, her parents uncovered a devastating rare genetic mutation with no cure in...

30/01/2026

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Market News

FDA Approves Breakthrough RRP Therapy

A groundbreaking therapy for a rare disease just got FDA approval without randomized trials. Could this change how we treat...

18/08/2025

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