The Trump administration’s funding cuts to the NIH could destroy a wave of approaching research breakthroughs

UK pharma group acquires Belgium’s EsoBiotec as it announces EU has approved Imfinzi lung cancer drug

Four children can now see shapes, find toys, recognise their parents’ faces and some can read and write

Dating, holidays, work: this genetic disease changes everything. Gene therapy could be a lifeline, if it’s accessible to all, says Tobi Oredein, founder and CEO of Black Ballad

We recommend a more proactive approach to identifying devices at risk and essential devices, increasing the use of exceptional market approvals, expanding the role of expert panels, engaging with the...

Voor het eerst in België hebben dokters een micro-pacemaker ingeplant bij een te vroeg geboren baby met een ernstige hartafwijking. De operatie gebeurde meteen na de geboorte. Het toestel is wereld...

Kendric Cromer, 12, is among the first patients to be treated with gene therapy just approved by the F.D.A. that many other patients face obstacles to receiving.

Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezac...

Campaigners hail decision to give thousands of sufferers access to new set of drugs known as ‘modulators’

Rare diseases most often emerge in childhood, yet getting a diagnosis can take up to nine years, and many children die by age 5.

At one point she feared she was experiencing a psychotic break; her husband wondered if she was secretly drinking.

Rare diseases pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the...

Lenmeldy’s $4.25 million price tag raises critical questions about patient access to a growing number of costly gene therapies.

Dutch immunologist Jacques Neefjes believes the drug aclarubicin, unavailable in Europe for 20 years, could have helped 100,000 people with a rare blood cancer

Progress in the quest to help progeria patients suggests that gene editing techniques may help treat other ultrarare conditions.

Orphanet Journal of Rare Diseases - In many countries worldwide orphan drug regulations are installed but only the United States of America and Japan have an orphan device regulation. For many...

Pharmacist Ian Soulairol discusses how 3d printing technology is evolving and what it means for clinical practice now and in the future.

The woman’s disorder was diagnosed when she was a child. Thirty years later, she decided to have more testing done.