No rare disease should go untreated—experts

WHILE the vast majority of rare diseases lack approved, specific treatments or cures, health experts emphasize the importance of regular consultations and treatments that not only manage symptoms but also prevent complications.

- CANDY P. DALIZON

“This situation is often a stumbling block forpatients and their families. Because there is no known cure, they may lose hope,” said Dr. Loudella Castillo, a child neurologist and neuromuscular specialist.

“Rare diseases should not be left untreated, no matter what the condition. As doctors, we guide patients and their families in terms of supportive care and treatment, while anticipating complications and monitoring their symptoms,” she continued.

Dr. Castillo was one of the speakers during a recent forum held in observance of Rare Disease Week 2026. AstraZeneca Philippines, togetherwith healthcare experts, government representatives, and patient advocacy groups, convened for an Agham Kapihan titled “More Than We Can Imagine: Amplifying Voices to Strengthen Equitable Rare Disease Care in the Philippines.” The forum called for earlier diagnosis, stronger care coordination, and improved implementation of existing policies for people living with rare diseases.

Rare diseases comprise a group of disorders affecting 400 million people globally, a population larger than those living with cancer and AIDS combined. Defined in the Philippines as conditions affecting one in 20,000 individuals, these diseases significantly diminish the quality of life for patients and place a heavy social and economic burden on their families.

“Individually, per condition, the number is low. But when you combine all these rare diseases, that’s a significant number,” said Dr. Melanie Alcausin of the University of the Philippines-National Institutes of Health. “That number translates to people, and people to families that are impacted by these conditions.”