As interest in rare disease drug development has grown in recent years, a couple of themes have emerged that are particularly ...

Continuing our rare disease theme, Catherine McLoughlin shares her thoughts on the orphan drug market and how pharma / biotech companies may change their focus to rare diseases in the future.

Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence ...

The University of Minnesota’s annual Rare Disease Day seminar this year, held at the end of February, explained how patients with rare diseases can utilize telehealth services nationally and ...

Guest Opinion. About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare ...

Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...

In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...

Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym ...

AstraZeneca PLC aims for $80 billion revenue by 2030 through strong growth in oncology, biopharmaceuticals, and rare diseases ...

In 2021, the Government of India established Centres of Excellence (CoEs) under the National Policy for Rare Diseases to ...

About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare Disease Summit ...