3 results
Primary Objective:Assess the safety and explore the biologically effective dose (BED) and/or the maximum tolerated dose (MTD) and/or the recommended Phase II dose (RP2D) of AZD4573 in patients with relapsed or refractory haematological…
Progesterone for Breast Development in Trans Women; Assessment of effects and safety -a pilot trial-
To explore the effects on breast development of addition of progesterone to the treatment with estradiol in trans women after vaginoplasty or orchiectomy. Secondary objectives include safety and patient satisfaction, mood, and sleep.
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…