Medical Science Liaison, PKU (phenylketonuria), Mid West / Mid Atlantic Region

Location:  remote, United States Category: Medical Affairs


BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options. BioMarin will continue to focus on advancing therapies that are the first or best of their kind.

BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in- class and best-in-class therapeutics that provide meaningful advances to patients who live with rare diseases. Come join our team and make a meaningful impact on patients’ lives.


The PKU (phenylketonuria) Medical Science Liaison (MSL or Sr. MSL) is a regional position supporting the scientific and business interests of BioMarin by cultivating current and future thought leaders in academic and clinical medicine at the local, regional and national level through in-depth, fair-balanced scientific discussions and interactions.

The MSL will support clinical trials through company-sponsored research, the initiation and development of Investigator Research proposals (IRs) and support of BioMarin registries, safety studies and other clinical programs. These thought leader interactions will complement or supplement the overall PKU product development plan. The MSL must possess in depth knowledge of BioMarin’s approved products, agents in the pipeline, and all associated disease state areas. The MSL must possess the capacity to coordinate and manage solo initiatives or partnerships with external partners independently or with a minimum amount of oversight. This position will report into the Global Medical Affairs department and work closely with the Commercial and the Clinical Teams.


MSL experience is required and experience in rare diseases is preferred. Territory will be focused in the Mid-West/Mid Atlantic



  • Develop and execute PKU scientific plans within their region to meet company-defined goals within corporate and regulatory guidelines for products both marketed and in development
  • Visit potential research sites to determine the site’s ability to conduct and accrue within clinical research studies and registries (i.e. patient volume, research experience, methodologies, competing studies)
  • Implementation and coordination of external research (Investigator Research) intended to support the clinical and scientific strategy for the PKU team
  • Identify and build mutually beneficial long-term relationships with key PKU Centers, investigators and key institutions to address current and future business needs. Support the clinical department by forwarding potential investigators for other BioMarin products in development. Maintain clinical and technical expertise in PKU therapeutic area; review scientific journals, attend scientific and key technical meetings and partner with medical, research and commercial teams
  • Conducts and analyzes literature searches to provide clinical insights for North American Medical Affairs
  • Demonstrate thorough understanding of both PKU clinical strategies and priorities
  • Deliver high quality, scientific and fair-balanced discussions and presentations on BioMarin products to physicians, NPs, dietitians and other key external customers
  • Provide medical support of BioMarin products during scientific symposia
  • Identify and recommend speakers for BioMarin’s Speaker’s Bureau and Advisory Boards
  • Assist in the site level management of the publication plan timelines
  • Provide clinical education support for internal customers
  • Proactively provides feedback on emerging clinical/competitive trends
  • Assure compliance with BioMarin operational guidelines



Required Skills:

  • Sr. Medical Science Liason:  5 or more years of relevant experience in clinical and 3 or more years as an MSL preferred
  • Medical Science Liason: 3 or more years of relevant experience in clinical and 1 or more year as an MSL preferred
  • Ability to quickly and easily learn new scientific and technical information as well as ability to interpret key information and translate into educational and research needs
    Excellent oral and written communication skills and interpersonal skills
    Excellent organizational skills and ability to manage a territory
  • A team player
  • Project management understanding and experience
  • Position will require 60-75% travel for field and home office obligations

Desired Skills/knowledge:

  • Experience in rare genetic diseases/PKU
  • Experience in product launch development and execution
  • Experience in product strategy and execution
    Knowledge of regulations impacting Medical Affairs and field activities in the pharmaceutical industry



  • MD, PharmD, PhD or equivalent degree preferred
    • Will consider master’s degree or advanced professional certification (e.g. NP) with extensive experience


  • This position will report into the Global Medical Affairs department and work closely with the Commercial and the Clinical Teams.