NAUTILUS EDUCATION | BETA PRODUCT “I mdefinitelya fighter, andwilltry and walk for as long as Ican.” researchers and funds devoted to them. With quiet National Institute of Health allocated $3.5 billion to stoicism, the Topics have accepted that modern medi- research orphan diseases. Yet some diseases are so rare cine may not have a solution for their daughters’ dis- that they remain stepchildren even among orphans. ease. Still, says Marijana, “Without hope, there’s no As a result, they receive little research attention and life.” funding. Neither do they fit the list of billable msur- Following a current grassroots trend in medicine, ance procedures. There’s no standard healthcare path many individuals with orphan diseases do not wait for to diagnosis, let alone treatment. Similar to the Topics, the medical industry to care about them. Facing long many patients go through an ordeal, which Maryana odds, they are forced to raise money to finda potential describes as “a blur,” only to find out that medicine cure themselves. But the Topics live by modest means. _ can’t help them. Marijana runs a daycare center and her husband and Orphan disease organizations, such as the National the childrens’ father, Niko, works for a lumber com- Organization for Rare Disorders and the Rare Disease pany. They are in no position to mount a quest. Foundation, encourage patients to take matters into But then there’s Michele Wrubel, 49, a stay-at-home their own hands. “Families have to advocate,” says Isa- parent from Connecticut who has calpainopathy. For — bel Jordan, chair of the Rare Disease Foundation. She years, Wrubel has been a passionate crusader for a encourages patients to form organizations, find new cure. Affluent and well connected, she doesn’t varnish methods of funding, and push for research. the truth about what it has taken to make the medical “Push for research” could be Michele Wrubel’s call- industry pay attention to her. “To make a difference in ing card. She wa