As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.

One in 10 Americans suffers from a rare disease, a statistic that casts the label “rare” in new light. It also drives the work of the National Organization for Rare Disorders, a coalition of ...

High clinical development costs and small target patient populations have often been presented as justifications for high prices of drugs to treat rare diseases. Historically, payers have accepted ...

Kendall Bryce, 25, has aquagenic urticaria - a rare condition which causes her skin to swell and break out in hives whenever she comes into contact with water. She says she feels pain whenever she ...

Rare diseases present unique challenges that demand our immediate attention and action — not only because of the financial and emotional burden they impose on families[i] but also because of the ...

The two brothers have Duchenne muscular dystrophy — a rare, inherited muscle-wasting ... gene therapy shows positive signs of slowing the disease's progression. Unfortunately, it is not an ...

Kendall Bryce, 25, has aquagenic urticaria, a rare condition which causes her skin to swell and break out in hives whenever she comes into contact with water. The mom of one, who is currently ...

A BRADFORD mum has expressed worry after her baby daughter was diagnosed with a condition only 220 people have in the world. 16-month-old Florence Violet, of Buttershaw, is one of 17 in the UK to have ...

For families with children facing rare medical conditions, hearing that treatment will require neurosurgery is an immensely overwhelming moment. Instantly, deep fear sets in and critical questions ...

Kendall Bryce, 25, has aquagenic urticaria – a rare condition which causes her skin to swell and break out in hives whenever she comes into contact with water. The mom-of-one, who is currently ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...