Posts

In his role, Reilly will continue to drive the rapid growth and innovation of health marketing services across dentsu.

Ipsen bolsters rare disease portfolio with acquisition of liver disease specialist Albireo.

Pfizer Inc. is exploring options for some early-stage treatments for rare diseases and cancer in a bid to focus on “high-impact” medicines and vaccines, the company said on Thursday.

For a decade, RPV has specialized in connecting clients with patients and family caregivers for a variety of research studies.

2023 marks the 40th anniversary of the Orphan Drug Act (ODA) and kicks off a year of renewed hope and promise for developing rare disease medicines.

Breaking down barriers in support of patients with rare diseases There are over 7,000 rare diseases and one in 10 worldwide have a rare disease. Over 675 diseases have some research and development targeting supportive care, treatment or a cure for these patients and with approximately only five percent of indications having a treatment available. […]

The agency’s top five represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

The company delayed the timeline for reporting key data from a trial of its cannabis-based drug to treat a rare genetic disorder, citing difficulties in enrolling patients amid a rise in respiratory illnesses.

If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.

The companies have initiated a research agreement supporting the development of new AI-ECG algorithm to help identify patients who may be at risk of cardiac amyloidosis.