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Mohamed Ladha, President and General Manager for Recordati Rare Diseases North America, discusses the company’s rare disease initiatives.

The U.S. branch of Recordati was established in 2013 with a focus on products to treat patients with rare inherited metabolic disorders including NAGS deficiency, cystinosis, and acute intermittent porphyria. In 2019, the company expanded into the endocrinology space, with products for diseases such as acromegaly, Cushing’s disease, Cushing’s syndrome, and Castleman disease. Most recently, Recordati has added hematological conditions to its pipeline/portfolio with a focus on cold agglutinin disease.

Mr. Ladha explains the unique and varying challenges of rare diseases. This often includes the frequency of misdiagnosis and lengthy time to a proper diagnosis, adequate knowledge and ability of physicians to treat and care for rare disease patients, the severity and chronic nature of such diseases, and educating patients on benefits of long-term treatment.

These challenges highlight the importance of raising awareness and education for rare diseases. This can lead to quicker and more accurate diagnosis and better decision making and approaches to patient care and treatment. Mr. Ladha is hopeful that in the coming years, advances in technology and the use of artificial intelligence may assist in identifying and aggregating metrics that can optimize and customize patient diagnosis and care.

Chapters:
Introduction 00:00
Recordati's Work in Rare Disease 1:02
How Rare Diseases Differ 4:48
Why Doctors Should be Aware of Rare Diseases 7:07
Recordati's Future 8:38