Patient description: The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy

Clinical trial eligibility criteria: eligible ages (years): 3.0-18.0,  Pontine Glioma Subjects must have radiographically proven diffuse intrinsic pontine glioma and confirmation of residual disease after initial therapy or at the time of recurrence/progression as confirmed by MRI of the brain Subjects must be age ≥3 years and ≤ 18 years Diffuse intrinsic pontine glioma with measurable disease after receiving radiotherapy either concurrent with or followed by ≤ 2 prior courses of chemotherapy Measurable disease as defined by: Measurable tumor >10mm by MRI Karnofsky performance status (PS) 60-100% (for patients > 16 years of age) OR Lansky PS 60-100% (for patients ≤ 16 years of age) Body surface area > 0.67 m2 and ≤ 2.21 m2 Life expectancy of at least 2 months A negative urine pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses) Acceptable liver function as defined by: 1. Bilirubin ≤ 1.5 times upper limit of normal 2. AST (SGOT), ALT (SGPT) and Alkaline phosphatase ≤ 2.5 times upper limit of normal Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age as follows  Concurrent anti-cancer therapy (chemotherapy, radiation therapy, surgery, immunotherapy, hormonal therapy, biologic therapy) other than the ones specified in the protocol. Patients must have discontinued the above cancer therapies for generally about 3 weeks (8 weeks for radiotherapy) prior to the first dose of study medication, as well as recovered from toxicity (to ≤ than grade 2 except for alopecia) induced by previous treatments Currently receiving another investigational medicinal product Uncontrolled concurrent illness including, but not limited to: 1. Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy 2. Diarrhea of any cause ≥ CTCAE grade 2 3. Psychiatric illness/social situations that would compromise patient safety or limit compliance with study requirements including maintenance of a compliance/pill diary 4. Any kind of malabsorption syndrome significantly affecting gastrointestinal function Pregnant or nursing female patients. NOTE: If a female patient becomes pregnant or suspects that she is pregnant while participating in this study, she should stop taking study drug and immediately inform her treating physician immediately Prior therapy with a Hedgehog inhibitor Unwillingness or inability to comply with procedures required in this protocol Serious nonmalignant disease (e.g., hydronephrosis, liver failure, or other conditions) that could compromise protocol objectives in the opinion of the investigator and/or the sponsor History of Congestive Heart Failure (CHF) or ventricular arrhythmia requiring medication