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We are committed to discovering and developing treatments for rare MC4R pathway diseases. This pathway plays a critical role in how the brain regulates hunger and energy expenditure (calorie burning). MC4R pathway function can be disturbed by certain gene variants or injury to the brain region known as the hypothalamus. Different from more common causes […]
We’re committed to advancing genetic understanding to fuel MC4R pathway research, disease knowledge, and a community of physician-scientists focused on learning more. Our unique DNA sequencing data platform Our approach to understanding MC4R pathway diseases is built upon a DNA sequencing infrastructure that includes genetic samples from approximately 80,000 people with severe obesity. (Each sample […]
Ambar, living with Bardet-Biedl syndrome, and her mother Mayra We are focused on developing treatments for diseases that impact the MC4R pathway. These include: POMC/PCSK1/LEPR deficiencies Rare biallelic variants of the POMC, PCSK1, and LEPR genes can result in deficiencies of certain proteins and enzymes that help the body regulate food intake, resulting in insatiable […]
The MC4R pathway plays a pivotal role in the underlying biology of many forms of severe hyperphagia and obesity. We are committed to using our growing understanding of this pathway to develop new therapeutic options…to relieve the substantial burden of living with these rare diseases. Alastair G. https://player.vimeo.com/video/805991350?h=08b3713786 Not all obesity is the same Rare […]
Popular searches MC4R IMCIVREE OBESITY Quick links Careers At Rhythm, we know our work makes an important difference in the lives of patients and their families around the world. Every employee contributes to the company’s goal of discovering new treatments for rare diseases that no other company is developing. Patients Rhythm is deeply committed not […]
Our mission To transform the lives of patients with rare neuroendocrine diseases by rapidly advancing care and precision medicines that address the root cause. Our vision Patients with rare neuroendocrine diseases are able to live their fullest lives and are transformed through optimal, stigma-free care. It takes great people to make a great company It’s the people […]
Mayra, a Bardet-Biedl syndrome caregiver, at Rhythm Rhythm is deeply committed not only to developing medicines for rare neuroendocrine diseases, but also to helping patients with these diseases receive the education, awareness, and access they need. Robert and Gabe, brothers living with Bardet-Biedl syndrome A patient and caregiver meeting at Rhythm Rhythm InTune: resources for […]
At Rhythm Pharmaceuticals, we are committed to our clinical trials program designed to develop innovative therapies for people with rare neuroendocrine diseases. These trials help us evaluate safety and efficacy of these potentially life-changing treatments. If you think you may be eligible, speak with your healthcare provider about the possibility of participating in one of […]
At Rhythm, we are dedicated to discovering therapies for rare neuroendocrine diseases. Our science is focused on developing therapies for patients with hyperphagia (insatiable hunger) and obesity caused by an impaired melanocortin-4 receptor (MC4R) pathway. We are also investigating treatments for other rare diseases, including congenital hyperinsulinism. The MC4R pathway Diseases caused by MC4R pathway […]
Rachel and her sons Wyatt and Carson, both living with Bardet-Biedl syndrome Rhythm Pharmaceuticals is a global biopharmaceutical company committed to transforming the lives of patients with rare neuroendocrine diseases by rapidly advancing care and precision medicines that address the root cause. Who we are We are a community-focused company, dedicated to healthcare providers, patients, and […]
