Author Archives for Daniel

Targeting the Epigenetic Drivers of Cancer

August 23, 20194:52 pm Published by Comments Off on Targeting the Epigenetic Drivers of Cancer

Epigenetics is the study of the regulatory system that controls how genes are turned on or off. In certain cancers,


Retrophin PKAN Therapy Fails in Late Stage Trial

August 22, 20196:44 pm Published by Comments Off on Retrophin PKAN Therapy Fails in Late Stage Trial

Rare Daily Staff Retrophin said that its late stage therapy for the rare neurodegenerative condition PKAN did not meet is


FDA Rejects Sarepta DMD Drug Because of Safety Concerns

August 20, 20195:02 pm Published by Comments Off on FDA Rejects Sarepta DMD Drug Because of Safety Concerns

Rare Daily Staff The U.S. Food and Drug Administration notified Sarepta Therapeutics that it would not approve its experimental exon-skipping


Passage Bio Launches GM1 Natural History Study

August 19, 20199:20 pm Published by Comments Off on Passage Bio Launches GM1 Natural History Study

Rare Daily Staff Passage Bio, which is developing a gene therapy for progressive neurological condition GM1 gangliosidosis, said it launched


FDA Approves Celgene’s Treatment for Rare Bone Cancer

August 19, 20199:18 pm Published by Comments Off on FDA Approves Celgene’s Treatment for Rare Bone Cancer

Rare Daily Staff The U.S. Food and Drug Administration approved Celgene subsidiary Impact Biomedicines’ Inrebic to treat adult patients with