JCR Pharmaceuticals Co., Ltd., a leader in therapies for rare and genetic diseases, has reached an important milestone in its ongoing collaboration with Alexion, AstraZeneca’s rare disease division. The achievement,…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
AstraZeneca, a global leader in pharmaceuticals, has taken a significant step into the rare disease arena in South Africa, aiming to improve diagnosis, treatment, and support for patients facing these…
Sanofi has announced a significant regulatory milestone for its investigational therapy SAR446523, a monoclonal antibody targeting GPRC5D, after the U.S. Food and Drug Administration (FDA) granted it orphan drug designation…
Biogen has achieved a significant regulatory milestone with the UK Medicines and Healthcare products Regulatory Agency (MHRA) granting approval for Qalsody (tofersen), a treatment specifically developed for a rare and…
A recently published case report has shed light on an uncommon cause of abdominal pain in adults: small bowel intussusception due to a benign tumor known as a lipoma. As…
Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
Nope, we're not joking! US SWAT teams (Special Weapons And Tactics, military weapons-equipped law enforcement teams) have been asked to step in to help with backlogged FDA applications to get experimental treatments designated as orphan…
As most readers of this site are probably aware, a “rare disease” is classified in the United States as one that affects fewer than 200,000 people. It’s estimated there are…
Bronchiectasis is a condition which damages the airways leading in and out of the lungs, stripping them of their mucus-clearing capabilities. Common variable immune deficiency (or CVID) is an immune…
For many people suffering from rare diseases, gene therapy offers the hope for relief and perhaps a cure for their particular condition. But while many studies are underway, to date, few drugs have…
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