Therapeutic Areas of Focus

Rare diseases affect about 1 in 12 Canadians or nearly 3 million people. These conditions are often genetic in origin. About 80% of rare diseases are caused by genetic changes.¹About CORD | Canadian Organization for Rare Disorders. (n.d.). https://www.raredisorders.ca/about/about-cord In 2023 Canada launched the National Strategy for Drugs for Rare Diseases to improve access to diagnosis, treatment and specialized care for those affected (Health Canada, The National Strategy for Drugs for Rare Diseases, 2024).²Health Canada. (2024, December 4). The National Strategy for Drugs for Rare Diseases. Canada.ca. https://www.canada.ca/en/health-canada/services/health-care-systems/national-pharmacare/strategy-drugs-rare-diseases.html

Among these diseases, we find hypoparathyroidism, a disease or syndrome due to a (transient or permanent) lack of PTH (Parathyroid Hormone) or PTH signaling. Symptoms include muscle cramps, kidney issues, and cognitive problems.³Bilezikian, J. P., Brandi, M. L., Cusano, N. E., Mannstadt, M., Rejnmark, L., Rizzoli, R., Rubin, M. R., Winer, K. K., Liberman, U. A., & Potts, J. T. (2016). Management of Hypoparathyroidism: Present and future. The Journal of Clinical Endocrinology & Metabolism, 101(6), 2313–2324.⁴Shoback, D. M., Bilezikian, J. P., Costa, A. G., Dempster, D., Dralle, H., Khan, A. A., Peacock, M., Raffaelli, M., Silva, B. C., Thakker, R. V., Vokes, T., & Bouillon, R. (2016). Presentation of Hypoparathyroidism: etiologies and clinical features. The Journal of Clinical Endocrinology & Metabolism, 101(6), 2300–2312. It’s considered permanent if it lasts over 12 months post-surgery.⁵Khan, A. A., Bilezikian, J. P., Brandi, M. L., Clarke, B. L., Gittoes, N. J., Pasieka, J. L., Rejnmark, L., Shoback, D. M., Potts, J. T., Guyatt, G. H., & Mannstadt, M. (2020). Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop. Journal of Bone and Mineral Research, 37(12), 2568–2585. A study reported that 70% of patients had hypoparathyroidism after surgery, while the remaining cases were due to autoimmune, genetic or unknown causes.⁶Khan, A. A., AbuAlrob, H., Punthakee, Z., Shrayyef, M., Werfalli, R. E., Kassem, H. A., Braga, M., Millar, A., Hussain, S., Iqbal, S., Khan, T., Paul, T., Van Uum, S., & Young, J. E. M. (2021). Canadian national hypoparathyroidism registry: an overview of hypoparathyroidism in Canada. Endocrine, 72(2), 553–561.

At Pendopharm, we address the needs of the rare disease community by bringing treatments to Canadian patients and supporting healthcare providers and patient organizations through medical education and patient support programs.

Gastroenterology focuses on the digestive system and its disorders. Digestive disorders are conditions that involve the gastrointestinal tract and encompass a variety of diseases ranging from mild to severe. Common digestive disorders include gastroesophageal reflux disease, irritable bowel syndrome, inflammatory bowel disease as well as some types of cancer such as colorectal cancer.⁷John Hopkins Medicine. Digestive Disorders. Overview. 2022. Available at: https://www.hopkinsmedicine.org/health/wellness-and-prevention/digestive-disorders. Accessed March 22, 2022.⁸National Library of Medicine. MedlinePlus. Digestive Diseases. April 1, 2022. Available at: https://medlineplus.gov/ency/article/007447.htm. Accessed March 22, 2022.

In Canada in 2022, colorectal cancer was expected to be the fourth most commonly diagnosed cancer.⁹Canadian Cancer Society. Colorectal cancer statistics. 2022. Available at: https://cancer.ca/en/cancer-information/cancer-types/colorectal/statistics. Accessed March 22, 2022. Fortunately, incidence rates are declining.¹⁰Canadian Cancer Society. Colorectal cancer statistics. 2022. Available at: https://cancer.ca/en/cancer-information/cancer-types/colorectal/statistics. Accessed March 22, 2022. The recent declines are likely due in part to increased use of colorectal cancer screening through tests such as fecal immunochemical test, sigmoidoscopy and colonoscopy, which can help detect and remove precancerous polyps and cancer, which can in turn reduce incidence.¹¹Canadian Cancer Society. Colorectal cancer statistics. 2022. Available at: https://cancer.ca/en/cancer-information/cancer-types/colorectal/statistics. Accessed March 22, 2022.

We are committed to the gastroenterology community through our support of medical education initiatives and relationships with physician and patient associations.

Sports medicine & orthopedics focuses on the musculoskeletal system, which encompasses bones, muscles, tendons, ligaments and soft tissues. Injuries, disease, and aging can cause pain, stiffness and other problems with movement and function of the musculoskeletal system.¹²Cleveland Clinic. Musculoskeletal System: Arthritis, Lower Back Pain, Bones, Muscles. December 11, 2020. Available at: https://my.clevelandclinic.org/health/articles/12254-musculoskeletal-system-normal-structure–function. Accessed March 22, 2022.

In Canada, approximately 3.9 million Canadians live with osteoarthritis, the most common type of arthritis.¹³Public Health Agency of Canada. Osteoarthritis in Canada. Highlights from the Canadian Chronic Disease Surveillance System. December 24, 2020. Available at: https://www.canada.ca/en/public-health/services/publications/diseases-conditions/osteoarthritis.html. In a 2009 survey of Canadians aged 20 years or older diagnosed with arthritis, 29% reported experiencing pain in their knee(s), while 29% had pain in both their knee(s) and hip(s).¹⁴MacDonald KV, et al. Symptom onset, diagnosis and management of osteoarthritis. Health Rep. 2014;25(9):10-7. 

Neurological disorders are conditions that affect the brain, spinal cord as well as the nerves found throughout the human body.¹⁵Merck Manual. Introduction to Symptoms of Brain, Spinal Cord, and Nerve Disorders. August 2021. Available at: https://www.merckmanuals.com/en-ca/home/brain,-spinal-cord,-and-nerve-disorders/symptoms-of-brain-spinal-cord-and-nerve-disorders/introduction-to-symptoms-of-brain-spinal-cord-and-nerve-disorders?query=Introduction%20to%20Symptoms%20of%20Brain,%20Spinal%20Cord,%20and%20Nerve%20Disorders. Accessed March 22, 2022. Common neurological disorders include epilepsy, a brain disorder that produces abnormal bursts of electrical activity in the brain and that affects about 300,000 Canadians of all ages and multiple sclerosis, a chronic autoimmune disease of the central nervous system which impacts the lives of an estimated 90,000 Canadians.¹⁶Public Health Agency of Canada. Epilepsy in Canada. December 9, 2019. Available at : https://www.canada.ca/en/public-health/services/publications/diseases-conditions/epilepsy.html. Accessed March 22, 2022.¹⁷MS Society of Canada. About MS. 2022. Available at: https://mssociety.ca/about-ms. Accessed March 22, 2022.

Heart disease is a major public health issue in Canada. It is the second leading cause of death, and one of the main causes of hospitalization.¹⁸Public Health Agency of Canada. Heart disease in Canada: Highlights from the Canadian Chronic Disease Surveillance System. July 18, 2017. Available at: https://www.canada.ca/en/public-health/services/publications/diseases-conditions/heart-disease-canada-fact-sheet.html. Accessed March 22, 2022. While prevention and education remain effective ways to help reduce the impact of this disease on Canadians, additional treatment options are essential.