A Rare Disease - Search News

FDA Is Approving More Drugs To Treat Rare Diseases

FDA smooths reviews of rare disease treatments

The Food and Drug Administration on Monday laid out a new pathway aimed at spurring novel individualized treatments targeting rare diseases. Why it matters: Officials said the draft rules would make it easier for industry to develop more types of promising new treatments for rare diseases,

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A New FDA Plan Could Transform Treatment Access for Rare Disease Patients

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FDA proposes new system for approving customized drugs and therapies for rare diseases

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FDA proposes new treatment approval pathway for ultrarare diseases

The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments addressing ultrarare diseases.

Becker's Hospital Review · 1d

FDA unveils pathway for ultra-rare disease therapies

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FDA fleshes out new roadmap for testing personalized therapies

10h

Rare disease isn’t rare—it’s a healthcare blind spot

In medicine, “rare” is often used to describe conditions that affect relatively few people. But when you work in healthcare long enough—especially at the very beginning of life—you realize rare diseases are not rare at all.

St. Louis Families Unite for Rare Disease Day 2026; Take Part Foundation Advances Critical Genetic Testing and Research

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and

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Your Stories: a Paralympic hopeful’s rare disease fight

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases.

10h

Local woman charts a path forward while living with a rare disease

AYVAKIT ® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 x 10 9 /L). It is not known if AYVAKIT is safe and effective in children.

University at Buffalo

Media Advisory: “Diagnose Earlier, Treat Smarter” is focus of UB’s annual Rare Disease Day for providers, patients and community partners

UB will will host its annual Rare Disease Day event from 1-5 p.m. on Friday, Feb. 27, in the Jacobs School of Medicine and Biomedical Sciences, 955 Main St., Buffalo.

FOX 2 Detroit on MSN

'I'm going to lose him': Michigan mother works to raise awareness as son endures late stages of rare disease

A mother from Belleville hopes to raise awareness about Lafora body disease, an ultra-rare disease her son is battling that has already claimed another one of her children.

Asianet Newsable on MSN

Are rare diseases always genetic? Myths, facts & what families should know

The definition of a “rare” disease is based on how often they occur in the population not by whether or not there is a genetic connection. Some rare diseases tend to run in families caused from a change in a single gene or in combinations of genes.

14h

Mstone Partners Healthcare Joins Global Efforts to Raise Awareness for Rare Disease Day

On the occasion of this Rare Disease Day, Mstone Partners Healthcare and our portfolio companies are proud to stand with the rare disease patient community, their families and caregivers, and researchers and advocacy groups to spotlight the tremendous need to bring new treatments for rare diseases.