A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

An international group of researchers led by Pompeu Fabra University has discovered the nanomachine that controls ...

Pancreatic cancer is a rare disease; however, its initially inconspicuous symptoms and aggressive nature make it one of the deadliest forms of cancer.

A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ultra-rare and life-threatening genetic disorder. Early findings suggest that a me ...

Australian researchers discover a targeted immunotherapy approach for myelofibrosis treatment, raising hopes for therapies ...

Maze Therapeutics uses its Compass genetics platform to advance kidney and obesity drugs, with MZE829 Phase 2 data due in ...

Ractigen Therapeutics is pleased to announce that the first patient has been dosed in the Phase II clinical trial of RAG-17, an innovative siRNA therapy targeting SOD1-mutated amyotrophic lateral ...

Published in the journal Blood, the research focuses on finding smarter ways to treat myelofibrosis by targeting the abnormal blood cells that drive the disease using immunotherapy, rather than just ...

Genetic treatments could be the key to unlocking some rare diseases. But bringing these medicines to patients involves ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to hereditary neuronal sodium ion channel mutationsAdolore's Kv7 voltage-gated po ...

The company made an even more dramatic claim the following month, when it announced it had created three dire wolves. These ...