News

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…

Chronic pain in adults with sickle cell disease (SCD), a frequent symptom of the disease, is associated with alterations in the connection of brain regions involved with pain perception, a study indicates. When SCD patients with chronic pain were compared to those without it, multiple differences were found…

Treatment with hydroxyurea since early infancy, starting at the age of one, is effective and safe at preventing sickle cell disease (SCD) complications, a follow-up study has found. The study “Prospective longitudinal follow-up of children with sickle cell disease treated with hydroxyurea since infancy” was published in journal…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Imara’s investigational oral inhibitor IMR-687 is well-tolerated, and shows signs of potential for lowering blood biomarkers of sickle cell anemia (SCA), early data from a Phase 2 trial show. The results were presented at the 24th Congress of the European Hematology Association (EHA), held recently in…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can markedly reduce both hemolysis — the destruction of red blood cells — and anemia in adolescents and adults with sickle cell disease (SCD), results from the Phase 3 HOPE trial show. Voxelotor also was found to significantly increase the…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

Howard University’s 6th Annual World Sickle Cell Awareness Day Symposium has its sights set on a cure for the inherited blood disorder. To be held June 19 — the 10th anniversary of World Sickle Cell Awareness Day — the free meeting is themed “Cure Sickle Cell Now — Patient Engagement.”…

Sevuparin, Modus Therapeutics' lead therapeutic candidate for managing painful vaso-occlusive crises (VOCs), failed to show clinically meaningful improvements in patients with sickle cell disease (SCD), Phase 2 clinical trial results show. Modus Therapeutics is part of Karolinska Development’s portfolio of pharmaceutical companies. Karolinska Development is a Sweden-based investment company focused on identifying and supporting breakthrough medical innovations developed by other companies. Sevuparin is an investigational drug with anti-adhesive and anti-inflammatory properties that has been developed to prevent the obstruction of blood vessels and restore normal blood flow, reducing the risks of VOCs, one of the most common and painful complications of SCD. The multicenter, randomized, double-blind, placebo-controlled, Phase 2 trial (NCT02515838) was designed to assess the safety and effectiveness of continuous intravenous infusion of sevuparin for the management of painful VOCs in SCD patients over the course of two to seven days, compared to a placebo. The trial's primary outcome was to assess the time of VOCs resolution after administering sevuparin. Secondary outcomes included treatment safety assessments, drug pharmacokinetic properties, mean change in pain intensity, duration of the most severe pain, and cumulative dose of opioids. (Pharmacokinetics is the study of how a drug is absorbed, distributed, metabolized, and eliminated from the body.) The study enrolled 144 SCD patients from multiple sites across Europe, the Middle East and the Caribbean. Unfortunately, according to statements from Karolinska Development, findings from the trial failed to show clinical benefits associated with the use of sevuparin. "It is disappointing that sevuparin did not demonstrate a meaningful clinical benefit in the Phase 2 study conducted by Modus Therapeutics, particularly as patients with sickle cell disease have no treatment options that can reduce the length of their vaso-occlusive crises or manage their pain, other than opioids. Modus will now consider options for further development of sevuparin in other indications where its multimodal action may have utility," Viktor Drvota, CEO of Karolinska Development, said in a press release. Karolinska said in it announcement that it will return with updated information as soon as Modus Therapeutics makes a final decision regarding the future direction of its development project involving sevuparin.