The ASH 2024 conference in San Diego featured updates on new treatments for rare diseases such as immune thrombocytopenia and sickle cell disease. Sanofi’s rilzabrutinib showed promising results in increasing platelet counts and reducing bleeding episodes in ITP patients. Novo Nordisk’s etavopivat pill reduced severe pain crises in sickle cell disease patients. Beam Therapeutics’ CRISPR-based therapy showed consistent results in increasing fetal hemoglobin levels in sickle cell patients. Additionally, research presented at the conference highlighted the viability of fertility preservation before curative therapy for sickle cell patients. Insurers may need to cover fertility services as more patients seek gene therapies for these diseases.
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ASH 2024: Roundup of news from first day of conference
