The American Society of Hematology (ASH) conference in San Diego saw several significant advancements in the treatment of rare blood disorders. Sanofi’s new oral medication, rilzabrutinib, achieved improved platelet counts and reduced bleeding episodes in patients with a rare autoimmune disorder that causes the body to attack its own blood-clotting platelets.
Novo Nordisk also made strides in treating sickle cell disease with their CRISPR-based therapy. Seven patients treated with the Beam Therapeutics’ BEAM-101 underwent a successful treatment, producing more than 60% fetal hemoglobin and significantly reduced levels of sickled hemoglobin.
Additionally, researchers found that fertility preservation before curative therapies can be safe for sickle cell patients, but only if they have access to experienced healthcare providers. New research from Johns Hopkins presented at ASH underscores the viability of this procedure for patients, despite potential complications such as pain crises after oocyte retrieval.
These advancements bring hope to patients suffering from rare blood disorders, and pave the way for future treatments that can improve their quality of life.
Source: https://www.statnews.com/2024/12/07/ash-2024-conference-sanofi-advances-drug-for-rare-platelet-destroying-disease