When adults withdraw from opioids, we understand that withdrawal symptoms follow. The lesser-known fact is that infants who are born dependent on drugs have horrible withdrawal symptoms, too. The difference is that they are defenseless.
Read moreBlood Cancer: Advancements in CAR-T Cell Therapies
September is Blood Cancer Awareness Month, a global event to help raise awareness of one of the world’s most prevalent and dangerous cancers. Accordingly, we examine advances in research that have the potential to improve treatments and outcomes.
Read moreMetachromatic Leukodystrophy (MLD) and Gene Therapy
Metachromatic Leukodystrophy (MLD) is a rare recessive genetic disorder caused by a mutation in the arylsulfatase-A (ARSA) gene, which normally breaks down sulfatide, resulting in the toxic buildup of lipids and other storage materials in cells in the white matter of the central nervous system and peripheral nerves.
Read moreCell & Gene Therapy Update: July 2023
June was a busy month for the FDA with the approval of two gene therapies, Elevidys (delandistrogene moxeparvovec-rokl) and Roctavian (valoctocogene roxaparvovec-rvox), and one cellular therapy Lantidra (donislecel-jujn). See more about these therapies.
Read moreFirst Allogeneic Cellular Therapy Approved for Type 1 Diabetes
On June 28, 2023, the U.S. Food and Drug Administration approved Lantidra, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes.
Read moreSickle Cell Disease: Hope for the Future
Every June 19, we observe World Sickle Cell Day to raise international awareness of sickle cell disease and the challenges patients and families face when handling the disease. Sickle cell disease is a group of inherited red blood cell (RBC) disorders that affect hemoglobin, the protein that carries oxygen through the body.
Read moreGene Therapy Update: SRP-9001 delandistrogene moxeparvovec for DMD
The FDA has pushed back the decision on the experimental gene therapy for Duchenne Muscular Dystrophy (DMD) until June 22.
Read moreMelanoma Vaccine Update
The FDA has granted breakthrough therapy designation for mRNA-4157 and Pembrolizumab combination for melanoma.
Read moreUnderstanding Chronic Kidney Disease
Thirty-seven million Americans, or one in every seven people, have chronic kidney disease (CKD). Surprisingly, however, 90% of these people are not aware they have this disease.
Read moreHumira Biosimilars Now Available in the US
On January 31, 2023, Amgen introduced a biosimilar for one of the pharmaceutical’s industry’s most lucrative products. Amjevita became first of nine biosimilar lookalikes scheduled to launch this year to compete with Humira (adalimumab).
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