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.
Researchers worked to better understand and manage patients with mild or moderate hemophilia, who can be an underrepresented and underserved population.
A group of US-based hemophilia experts summarized unmet needs in mild to moderate hemophilia and provided suggestions for healthcare professionals to optimally manage their patients with this challenging condition.