Alas, porphyria

For over two decades, treatment of acute porphyrias has relied on hemin infusions that restore heme levels, but the therapy is slow to work, has side effects and is expensive. Now, a team led by Alnylam Pharmaceuticals Inc. has created an siRNA therapeutic that reduces disease symptoms in mice.¹

However, to really improve patient care, the company will also need a strategy to raise awareness and combat misdiagnosis of these rare disorders...