A recent study highlights the multidisciplinary management approach for an older adult with vascular Ehlers-Danlos syndrome and multiple autoimmune comorbidities. This case underscores the complexity of treating patients with rare diseases and associated conditions.

You may report side effects to U.S. FDA at 1-800-FDA-1088. ... Celiprolol is Zevra’s investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the U.S. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. toll-free at 1-844-600-2237 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. Zevra recently restarted enrollment in the DiSCOVER trial, a Phase 3 trial being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. Enrolled four patients in the event-driven Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehlers-Danlos Syndrome during Q2 2026, bringing the total number of enrolled patients to 66, with a total of three confirmed events. The Company expects to continue engagement with the U.S. Food and Drug Administration (FDA) in the second half of this year to explore pathways to accelerate clinical development.

A study identifies rare type 1 collagen variants in early-onset bicuspid aortic valve disease, revealing clinical and genetic overlaps with Ehlers-Danlos syndrome and osteogenesis imperfecta. This research enhances understanding of these connective tissue disorders.

A recent cross-sectional study investigates the oral manifestations of non-vascular Ehlers-Danlos syndrome, providing new insights into the condition's impact on oral health. This research contributes to the understanding of the syndrome's broader implications for patient care.

A personal account highlights the struggles of living with multiple rare diseases, including Hashimoto’s disease, rheumatoid arthritis, fibromyalgia, and postural orthostatic tachycardia syndrome (POTS). The individual faces significant medical costs and challenges in obtaining disability support, emphasizing the need for greater awareness and advocacy for those with complex health conditions.