Rare disease experience

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 syndromeIgA nephropathy
Immune thrombocytopeniaSevere aplastic anemia
Psoriatic arthritisAnkylosing 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 cancerExtensive-stage SCLC
Metastatic pancreatic cancerTriple negative breast cancer
Rare target cancers
ALK+ NSCLCBRAF+ NSCLC and melanoma
Anti-CEACAM5 NSCLC (failed trial)