Rare Disease News and Research

RSS
New sickle cell disease poll dispels long-held perceptions of African Americans

New sickle cell disease poll dispels long-held perceptions of African Americans

Researchers precisely describe effects of frequent genetic mutation in congenital hyperinsulinism

Researchers precisely describe effects of frequent genetic mutation in congenital hyperinsulinism

Without price breaks, rural hospitals struggle to stock costly, lifesaving drugs

Without price breaks, rural hospitals struggle to stock costly, lifesaving drugs

Wearable technology launched by egg health allows first person patient insight

Wearable technology launched by egg health allows first person patient insight

Insmed reports top-line data from Phase 3 study of new treatment in patients with NTM lung disease

Insmed reports top-line data from Phase 3 study of new treatment in patients with NTM lung disease

Combination immunotherapy as second or third line treatment of mesothelioma shows promising results

Combination immunotherapy as second or third line treatment of mesothelioma shows promising results

Coimbra Pediatric Hospital selects Congenica's Sapientia software platform for In2Genome project

Coimbra Pediatric Hospital selects Congenica's Sapientia software platform for In2Genome project

FDA grants accelerated approval to new treatment for children with Chagas disease

FDA grants accelerated approval to new treatment for children with Chagas disease

New antibodies may halt key pathological mechanisms across several neurological diseases

New antibodies may halt key pathological mechanisms across several neurological diseases

Pfizer announces launch two new digital innovations to help people living with hemophilia

Pfizer announces launch two new digital innovations to help people living with hemophilia

What happens if you’re forced to switch health plans when you’re sick?

What happens if you’re forced to switch health plans when you’re sick?

Researchers uncover how mutations in single gene lead to rare kidney and liver disease

Researchers uncover how mutations in single gene lead to rare kidney and liver disease

Bionic vision restoration: could future technologies restore sight?

Bionic vision restoration: could future technologies restore sight?

Insurers can bend out-of-network rules for patients who need specific doctors

Insurers can bend out-of-network rules for patients who need specific doctors

Blood biopsy helps identify targetable genetic alterations in patients with cancer of unknown primary

Blood biopsy helps identify targetable genetic alterations in patients with cancer of unknown primary

Understanding the rise in NTM infections

Understanding the rise in NTM infections

Lab-grown miniature blood vessels mimic rare Hutchinson-Gilford Progeria Syndrome

Lab-grown miniature blood vessels mimic rare Hutchinson-Gilford Progeria Syndrome

Researchers unravel genetic characteristics that increase risk of developing Tourette syndrome

Researchers unravel genetic characteristics that increase risk of developing Tourette syndrome

Hospitals slashed use of two heart drugs after huge price hikes

Hospitals slashed use of two heart drugs after huge price hikes

Scientists propose therapeutic strategy to suppress formation of hereditary bony tumors

Scientists propose therapeutic strategy to suppress formation of hereditary bony tumors