World Gaucher Disease Day is observed every year on October 1st. On this day, patients, families, healthcare professionals, researchers, associations, and industry partners come together to raise awareness about this rare disease across the globe. In 2025, the International Gaucher Alliance (IGA) ...

World Duchenne Muscular Dystrophy (DMD) Day is observed on September 7, raising awareness of this rare, genetic, and progressive disease that most commonly affects boys. The aim of this day is to highlight the importance of early diagnosis and access to therapies, provide support to families, and encourage further research and development of new...

Gastroschisis Awareness Day is observed every year on July 30th to raise awareness about this rare but serious congenital condition affecting newborns. Gastroschisis is a defect of the abdominal wall through which abdominal organs, most commonly the intestines—and sometimes others—protrude outside the baby's body.

August is Spinal Muscular Atrophy (SMA) Awareness Month – a time to shine a light on this group of rare but serious genetic diseases that cause the progressive loss of skeletal muscle due to degeneration of motor neurons in the spinal cord and brainstem. SMA leads to progressive muscle weakness, ...

World Scleroderma Awareness Day is observed on June 29 to raise awareness of this rare autoimmune disease that affects connective tissue, skin, and internal organs. The day also highlights the importance of early diagnosis, patient support, and medical research. Scleroderma, also known as systemic sclerosis, is a rare autoimmune connective tissue disease ...

International Phenylketonuria (PKU) Awareness Day is observed annually on June 28 to raise awareness about this rare but serious inherited metabolic disorder. The day emphasizes the importance of early detection, timely treatment, and lifelong adherence to a low-phenylalanine diet in order to prevent permanent damage to the brain and overall development.

International Dravet Syndrome Awareness Day is observed every year on June 23, with the goal of raising awareness about this rare and severe form of epilepsy, as well as providing support to those affected and their families. Dravet Syndrome (DS), also known as Severe Myoclonic Epilepsy of Infancy (SMEI)

World Motor Neurone Disease (MND) Awareness Day is observed every year on June 21. The aim of this day is to raise awareness about motor neurone diseases, such as Amyotrophic Lateral Sclerosis (ALS), Progressive Bulbar Palsy, Progressive Muscular Atrophy, Primary Lateral Sclerosis, and Progressive Pseudobulbar Palsy.