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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.

Global Blood Therapeutics (GBT) announced the five organizations that will receive more than $200,000 worth of grants through its new Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL). ACCEL was launched in February by the clinical-stage biopharmaceutical company with the goal of improving the…

Prominent sickle cell advocate and philathropist Howard Woolley has won a Sickle Cell Outstanding Service Award, presented recently at the 2019 Roland B. Scott Memorial Symposium. Themed “Preparing the next generation of advocates for sickle cell disease healthcare,” the symposium, held at Howard University, featured a…

Robert and Tanique Mitchell still remember the moment they found out that their granddaughter, Kiarra Roseburgh, would be born with sickle cell disease (SCD). “We realized that we needed to prepare, and we made two goals as a family: to provide emotional support for Kiarra and to empower ourselves…

IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). It’s currently being evaluated in a Phase 2a trial in the U.S. and U.K. The Fast Track process is meant to get new therapeutics that treat…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Novartis‘ Solutions to Empower Patients (STEP) program will fund five nonprofit organizations that address some of the most pressing concerns in the sickle cell disease (SCD) community. Nearly $250,000 will be divided up among patient advocacy organizations and research institutions. Started in 2017, the STEP program funds innovative…

The Phase 2 STRONG-SCD clinical trial evaluating olinciguat as an investigational therapy for sickle cell disease is seeking participants. Olinciguat, being developed by Cyclerion Therapeutics (a spin-off of Ironwood Pharmaceuticals), is an investigational oral therapy that stimulates an enzyme called soluble guanylate cyclase (sGC), known to play a key…