Patient Advocacy

Patient Advocacy

Acer Therapeutics is a pharmaceutical company focused on the acquisition, development, and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. We are committed to supporting the patients and families that need it the most.

Patient support is at the core of everything we do at Acer. 

In support of that, Acer is dedicated to the following principles that serve as a foundation for interactions with rare disease stakeholders: 

  • Make a difference by bringing value to patients and caregivers 
  • Foster and sustain relationships with patient advocacy organizations 
  • Elevate the voice of the patient 
  • Be transparent in communications and business practices
  • Demonstrate compassion, ethics and integrity 

In the U.S., OLPRUVA™ (sodium phenylbutyrate) is indicated for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various other inborn errors of metabolism, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and Post-traumatic Stress Disorder (PTSD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. These product candidates are under clinical investigation and their safety and efficacy have not been established.  There is no guarantee that any of these products will receive health authority approval or become commercially available for the uses being investigated. 

If you are an advocacy organization interested in Acer Therapeutics, or if you are interested in sharing your story and getting involved with our current patient support programs, contact us at

Advocacy Partners and Professional Organizations:

The National Urea Cycle Disorders Foundation is a non-profit organi-zation dedicated to the identification, treatment and cure of urea cycle disorders. NUCDF is a nationally-recognized resource of information and education for families and healthcare professionals. The organization was formed in 1988 by a handful of parents whose children were affected. The Foundation is operated and supported by the volunteer efforts of families with children suffering from UCDs and is a supportive network of families, friends, and medical professionals. Its Vision is a world in which no child or adult will perish from UCD. Its Mission is to save and improve the lives of all those affected by urea cycle disorders.

UCD Foundation strives towards awareness, (medical and personal), education, and bringing hope to UCD friends and families. Its desire is to bring all together and continue Connecting Families all over the world.

The MSUD Family Support Group is a non-profit volunteer-run organization serving families and professionals worldwide. Founded in 1982 when a group of families gathered together for mutual support and shared information, our organization continues to grow and develop resources to meet our goals of support, education, expanded research, and advocacy.

The VEDS Movement mission is to save lives and improve the quality of life of individuals with Vascular Ehlers-Danlos Syndrome (VEDS). By pursuing the most innovative research, educating the medical community, general public and affected individuals, and providing support to patients, families, and caregivers, we can charge forward and improve the outcomes for those living with VEDS.

By connecting researchers, communities, and business leaders, the DEFY Foundation raises research funding and community awareness in support of patients and families impacted by Vascular Ehlers Danlos Syndrome. Learn more at

Annabelle’s Challenge is a UK charity for Vascular Ehlers-Danlos Syndrome. The charity raises funds for research, awareness campaigns and offers support and advice for people living with the condition. The charity also works closely with the NHS Ehlers-Danlos National Diagnostic Service with clinics in Sheffield and London. For more information visit

The Ehlers-Danlos Society is a nonprofit organization serving the global community to affect change and progress in the world of Ehlers-Danlos syndrome (EDS). Their mission is for all people with Ehlers-Danlos Syndrome to have their right for an early diagnosis, good management, respect, and recognition for their condition. The society is involved with:

  • Collaborative research
  • Organizing medical symposia and conferences
  • Bringing together and uniting the EDS community
  • Producing reliable, up-to-date medical literature
  • Building and uniting EDS support groups and charities from around the world
  • Giving HOPE to all those whose lives are affected by Ehlers-Danlos syndrome

Find out more at their website

FIGHT vEDS is a non-profit started by a vEDS patient and physician. The organization’s mission is to (1) provide patient-friendly information about vEDS, (2) help people quickly and affordably get tested for vEDS, and (3) connect vEDS patients with the right care team and patient support resources. Learn more at

The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 250 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

NORD serves the rare disease community, including patients and their families, patient organizations, researchers, medical professionals, and companies developing orphan products. They also work closely with many government agencies, most notably the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). NORD programs are focused on improving the lives of individuals and families affected by rare diseases. Find out more at their website

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. The group believes it “can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.” Learn more at

Global genes is an organization made up of advocates, partners and collaborators who are working to eliminate the daily challenges for 1 in 10 people affected by rare disease. The grou seeks to empower patients, build communities & drive forward momentum for rare disease globally. Find out more at their website

NIH Genetic and Rare Diseases Information Center

The Genetic and Rare Diseases Information Center (GARD) is a program of the National Center for Advancing Translational Sciences (NCATS) and is funded by two parts of the National Institutes of Health (NIH): NCATS and the National Human Genome Research Institute (NHGRI). GARD provides the public with access to current, reliable, and easy-to-understand information about rare or genetic diseases. Learn more at