A Global Leader for Rare Diseases

The college has a long history of leadership in addressing the challenges of rare diseases and orphan drug development. Over the years, that leadership has extended from Minnesota to India and across the globe.

Ramaiah Muthyala and Marilyn SpeedieIt’s taken decades of work and a commitment from researchers, patients, policy makers and clinicians to begin to effectively address the challenges associated with orphan drug discovery. The college’s leadership in finding treatment options for rare diseases began under Larry Weaver, who served as dean from 1966 to 1984.

“Dean Weaver had a passion for orphan drugs and worked diligently to get the Orphan Drug Act implemented after it was approved by Congress in 1983,” said Dean Emeritus and Professor Marilyn Speedie.

Weaver’s passion didn’t stop there

“When the college was establishing a series of endowed chairs in the mid-90s, he advocated for one of those chairs to be dedicated to orphan drug development,” said Speedie.

Today, Professor Jim Cloyd holds the Lawrence C. Weaver Endowed Chair and serves as the Director of the Center for Orphan Drug Research.

In 1994 Weaver also was involved in the founding of Minnesota-based Orphan Medical.

“Larry Weaver accomplished a great deal nationally by bringing attention to rare diseases and the need for orphan drug development,” said Speedie.

In the U.S. there are more than 7,000 rare diseases affecting 25 million Americans.

“The power of rare diseases is that when you cumulate all of the people who have rare diseases, it’s a huge number of people affected,” said Speedie.

From Minnesota to global

Fast forward to 2018 and the college’s leadership in orphan drugs has extended across the globe. Research Associate Professor Ramaiah Muthyala is the founder, president and CEO of the Indian Organization for Rare Diseases (IORD) — an umbrella organization representing all rare diseases and patients in India. Its mission is to raise the awareness of rare diseases in India, advocate for public policy and promote orphan drug development.

When Muthyala first came to the University, he joined the Center for Drug Design as senior associate director. After five years, he moved to the Center for Orphan Drug Research. While the orphan drug industry was progressing in the United States, the pharma industry for orphan drugs was nonexistent in many countries. As awareness spread, he launched a personal mission to raise awareness in India and other developing countries.

“In India, parents are often helpless when they have a child with a rare disease; the patient does not have a voice like patients do in the United States,” said Muthyala. “India supplies 25 percent of the world’s pharmaceuticals; however, there are no incentives for the pharmaceutical industry to develop drugs for rare diseases.”

In 2016, Muthyala organized the Indo-U.S. Rare Diseases Conference held on the University of Minnesota campus to increase awareness and encourage drug development collaborations. Minnesota Governor Mark Dayton proclaimed Nov. 16 of that year as Rare Disease Awareness Day.

“What Ramaiah has done is with the encouragement of the college, but he’s done this on his own through his leadership and commitment,” said Speedie. “He has been remarkably successful in India by bringing attention to rare diseases and his work has extended to a global awareness with the United Nations, the World Health Organization through Rare Diseases International and UN NGO committee for Rare Diseases and beyond.”

In the U.S. there are more than 7,000 rare diseases affecting 25 million Americans.