Clinical Trials and Drug Approval

FDA 101

Deborah Miller and Salina Prasad of the US Food & Drug Administration provide an overview of the agency and discuss patient advocacy as it relates to the FDA.

The Clinical Trial: Real-Life Experiences (2013 MDF Annual Conference)

Pat Furlong, CEO of Parent Project Muscular Dystrophy, will share the experiences of Duchenne community members who have participated in clinical trials.

The U.S. Drug Testing & Approval Process (2013 MDF Annual Conference)

Dr. Doug Kerr, M.D., Ph.D., of Biogen-Idec, describes the U.S. process for taking a drug from testing to approval.

Challenges of DM

People living with myotonic dystrophy have a frank discussion about the biggest challenges facing their lives.

Myotonic Dystrophy News

January 25, 2023

In Remembrance of Dad: Reynold Wantseng Wang

Written by his son, Dr. Eric Tzy-Shi Wang (王滋熙). My dad had lived with symptoms of myotonic dystrophy for over 30 years. After college, with the encouragement of friends and colleagues, I decided to focus my training and career on better understanding the molecular mechanisms of DM, and it had always been an ambitious goal of mine to find a medicine for my dad. Thus, my dad’s journey ultimately served as a guidepost and motivating factor for my life’s work.

January 25, 2023

Congress Ensures DM Remains Eligible For PRMRP Research Funding For Sixth Year in A Row

DM Researchers Awarded Record $8.8 Million in New Awards In Fiscal Year 2022 Four DM researchers submitted grant applications and two researchers won awards. This represents an excellent 50% success rate for DM! Details on the researchers and research topics will be announced soon. Read more about this increcdible funding opportunity and how you can become a PRMRP Consumer Reviewer!

January 25, 2023

Welcome Sofia Olmos, PhD, Myotonic Dystrophy Family Registry Coordinator

We are overjoyed to welcome Sofia Olmos, PhD, to the team! Sofia’s passion for science, as well as increased direct involvement in patient care communities, makes her a perfect fit for a role that requires both emotional intelligence and a comprehensive scientific knowledge of data storage practices, evaluation, and analysis.

January 25, 2023

Community Interview: Glenda Winson, Guillermo Zubillaga, and Jan Jaffe, New York City MDF Support Group Facilitators

It would be challenging to exist among the fast-paced lifestyle of Manhattan and surrounding boroughs. That is why it is important, especially as a person living with DM, to have support. One place we can seek out that mutual aid is within a support group. And in New York City, look no further, because Glenda, Guillermo, and Jan are the team of cheerleaders you need in the Big Apple.

January 25, 2023

Community Interviews: Jodie Howell & Samantha Welsh, Virginia State MDF Support Group Facilitators

Consciously dedicating hours of their week to support the myotonic dystrophy community, these support group facilitators have decided that they have a higher capacity to contribute. I am quite consistently taken aback by people like Jodie Howell and Samantha Welsh, who are actively involved in their own communities, but have determined that they have a greater threshold to give.

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Clinical Trials and Drug Approval

FDA 101

The Clinical Trial: Real-Life Experiences (2013 MDF Annual Conference)

The U.S. Drug Testing & Approval Process (2013 MDF Annual Conference)

Challenges of DM