Rare Diseases


Rare Diseases Report 2022

The paradox of Pompe disease

For nearly 2 decades, patients with even the most severe genetic mutations have been surviving on therapy. But clinicians must now contend with...

Rare Diseases Report 2022

Myasthenia gravis: Finding strength in treatment options

Although treatment might have once been considered stagnant, newer expert consensus and novel research are generating optimism for innovative...