An infusion of GENV-HEM results in endogenous expression of factor Va, which could improve homeostasis in patients with factor VIII or IX deficiency.
Hematologist Federico Stella, MD, says restrictive diets for patients in the hospital recovering from stem cell transplants are unnecessary.
Danny’s Dose Alliance aims to “change current outdated emergency treatment protocols that affect the proper care of special needs medical patients.”
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Generation Bio is working to “create the next generation of gene therapy” that will enable the redosable, durable expression of missing proteins.
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.
As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older—including the country’s 30 million or so rare disease patients—to get vaccinated quickly.