Qfitlia (fitusiran) is the first therapy for both hemophilia A or B, with or without inhibitors, available in the United States.

The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia ...

Experimental drug NU-9—a small molecule compound approved by the U.S. Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS)—improves ...

The National Alliance on Mental Illness (NAMI) released the results of a recent poll that asked Americans how they felt about proposed cuts to Medicaid. 1 According to the results, Americans are ...

The delay dovetails with moves by Republican lawmakers in at least 7 states to ban or limit mRNA vaccines. HealthDay News — The US Food and Drug Administration (FDA) has delayed the full ...

In a report released today, Emmanuel Papadakis from Deutsche Bank maintained a Hold rating on Sanofi (SNYNF – Research Report), with a price target of €100.00. The company’s shares closed ...

Sanofi’s attempt to take on Roche with a ground-breaking approach to treating haemophilia has hit a setback after the company voluntarily placed its late-stage trials of fitusiran on hold ...

The bleeding disorders community reminds me of that massive oak tree.

Fitusiran, which is in phase III clinical trial for hemophilia; cemdisiran that is in phase III clinical trial for Myasthenia Gravis and paroxysmal nocturnal hemoglobinuria, and geographic atrophy ...

Meanwhile, Sanofi is also hoping to file for approval of Alnylam-partnered RNA interference therapy fitusiran for haemophilia A and B this year, which works by suppressing antithrombin (AT), a ...