Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
Telemedicine, or the ability to conduct some physician consultations online, is having its moment. Envisioned by pioneers as the next revolution in promoting medical access, its popularity soared during the COVID-19 pandemic, when movement controls were initiated in many parts of the world. Today, regulators, physicians, and patients agree: telemedicine is here to stay. The…
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Myasthenia gravis (MG) is a complicated disease that takes an average of 2 years and 3 to 4 physicians to diagnose, expert Henry Kaminski, MD, said.
Meridith O’Connor, who has myasthenia gravis, founded The Merit Option to help other patients manage life with the disease.
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
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.
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