News

A team at the Case Western Reserve University has been awarded up to $3.7 million from the National Heart, Lung, and Blood Institute (NHLBI) to conduct early studies of emerging gene therapies for sickle cell disease (SCD). Led by Umut Gurkan,…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Forma Therapeutics‘ FT-4202 for the treatment of sickle cell disease (SCD) This designation is given to investigational therapies intended to treat rare conditions (those that affect fewer than 200,000 people in the U.S.). It provides companies…

The nonprofit HealthWell Foundation launched a new fund to help people with sickle cell disease afford their treatments. Awards will provide up to $10,000 over 12 months to eligible patients being treated for sickle cell in the United States. To qualify, patients must have health insurance that covers…

The Phase 2 STRONG-SCD trial evaluating olinciguat as a potential oral therapy for sickle cell disease is completely enrolled, Cyclerion Therapeutics said. Seventy patients with stable disease were recruited, and topline results are expected between July and September. The company is not expecting the supply of…

The Sickle Cell Disease Association of America (SCDAA) has announced the extension of its ongoing COVID-19 Emergency Fund Campaign to meet the needs of people with sickle cell disease (SCD) during the COVID-19 pandemic. The initial goal of the fundraiser was to raise $100,000 in 10…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Global Blood Therapeutics (GBT) is giving $250,000 to support the U.S. sickle cell disease (SCD) community during the COVID-19 pandemic. Its newly created GBT Community Fund will award a total of $150,000 in grants to non-profit organizations supporting the needs of  SCD patients and their families during…

The U.S. Food and Drug Administration (FDA) has cleared the start of a Phase 1/2 clinical trial testing a genome editing-based therapy, known as OTQ923, in adults with severe complications of sickle cell disease (SCD). OTQ923, developed by Novartis and Intellia Therapeutics, uses the CRISPR/Cas9 genome-editing technology.

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…