Trial of Gene Therapy for Sickle Cell Disease Halted
Two patients participating in the study developed cancer years after treatment
Sickle Cell Disease
MPR Weekly Dose Podcast Episode 57
This week we cover a new treatment submission to the FDA for the prevention of diabetes; The launch of a bupivacaine implant to treat post op pain; The DEA schedules a newly approved opioid; The FDA classifies over 200 drugs as essential and wants manufacturers to ramp up production; And more news on rifamixin and other drugs that are in the pipeline.
Endari Label Updated With Benefit Statement, Instructions for Use
Endari (L-glutamine; Emmaus Medical) is an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients aged 5 years and older.
Rifaximin Gets Orphan Drug Status for Sickle Cell Disease
Rifaximin is believed to reduce VOCs in patients with sickle cell disease through microbial modulation.
Siklos Now Available in 100mg Scored Tablets
Siklos is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in children with sickle cell anemia.
Medical Marijuana Tied to Fewer Admissions in Sickle Cell Disease
But no change in opioid use observed for those receiving medical marijuana certification
Novel Pyruvate Kinase Activator Gets Orphan Drug Status for Sickle Cell Disease
The Food and Drug Administration (FDA) has granted Orphan Drug designation to FT-4202 (FORMA Therapeutics) for the treatment of sickle cell disease (SCD).
New Drug Product: Oxbryta
This slideshow shows drug information for Oxbryta. Click here for the complete Oxbryta monograph.
Gene Therapy for Sickle Cell Disease Receives Orphan Drug Designation
The Food and Drug Administration has granted Orphan Drug designation to ARU-1801 (Aruvant) for the treatment of sickle cell disease.
MPR Weekly Dose Podcast: Episode 15
Back from our Thanksgiving break, this week we talk about the new approval for sickle cell disease; the re-introduction of a product that was taken off the market in the 60’s; The first ready-to-use phenylephrine injection; A new ultrasound enhancing agent; And an approval for arthritis.
