From neurologic to hematologic diseases
ALS was the first rare disease I worked on. That was way back when Rilutek launched. I remember tears rolling down my cheeks as I read patients’ and their care partners’ stories. The devastation of the disease was overwhelming. That was almost 30 years ago and they’re just now finally making breakthroughs in the treatment of this fatal disease.
Every rare disease I’ve worked on has the same impact on me. Often, they are disabling, debilitating, and/or deadly diseases with little to no major treatments. So, when breakthroughs do happen or when patients and care partners get more support and recognition, it makes me feel proud of the work I do.
Please see my category experience listed below.
| Rare diseases | |
| Amyotrophic lateral sclerosis (ALS) | Duchenne muscular dystrophy (DMD) |
| Fragile X syndrome | IgA nephropathy |
| Immune thrombocytopenia | Severe aplastic anemia |
| Psoriatic arthritis | Ankylosing spondylitis |
| Desmoid tumors | |
| Hematologic rare cancers | |
| Acute lymphoblastic leukemia (ALL) | Acute myeloid leukemia (AML) |
| Myelodysplastic syndromes (MDS) | Myelofibrosis |
| Chronic myeloid leukemia (CLL) | |
| Additional rare cancers | |
| Low grade serous ovarian cancer | Extensive-stage SCLC |
| Metastatic pancreatic cancer | Triple negative breast cancer |
| Rare target cancers | |
| ALK+ NSCLC | BRAF+ NSCLC and melanoma |
| Anti-CEACAM5 NSCLC (failed trial) | |