Solving the Rare Disease Equation - Dr. Alaa Hamed, Global Head of Medical Affairs, Rare Diseases at Sanofi

11/30/22 • 24 min

As we continue our focus on rare diseases on Raise the Line, we’re delighted to be joined by Dr. Alaa Hamed, Global Head of Medical Affairs, Rare Diseases at Sanofi, one of the leading pharmaceutical companies in the world. Although most well known for their focus on lysosomal storage disorders including Gaucher and Pompe disease, Dr. Hamed and his team at Sanofi are also working in adjacent disease spaces depending on the systems affected. “For example, the lysosome in Pompe disease affects the neuromuscular tissues, so we have a neuromuscular disorder interest as well.” In their discussion, Dr. Hamed and host Shiv Gaglani also touch on the efforts Sanofi is making to shorten the diagnostic odyssey for rare disease patients, including building more disease awareness and greater global infrastructure. “From the inception, we thought that having universal access is a key part of the rare disease equation.” You’ll also learn about the challenges of drug development, the importance of maintaining policy incentives to focus on rare diseases, and where innovation is needed most to advance outcomes for patients.

Mentioned in this episode: https://www.sanofi.com/

If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Mentioned in this episode: https://www.sanofi.com/

If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Previous Episode

Transforming the Way Radiologists Learn - Daniel Arnold, CEO of Medality

“Imaging has really become the tip of the spear of the patient journey,” says Daniel Arnold, CEO of Medality. In order to train future radiologists in this critically important and complex specialty, and keep current practitioners on top of their game, Arnold and his team are on a mission to transform the way radiologists learn by offering an online solution that mimics practicing radiology in the field. “Our goal is to make it easy for radiologists to learn a new subspecialty in just five minutes per day.” In his conversation with host Shiv Gaglani, Arnold touches on how Medality (formerly MRI Online) is connecting radiology practices with people who have the skills they need most. The two also discuss the importance of getting imaging diagnoses correct the first time, why radiologists can't just rely on what they learned in residency and fellowship, and the impact of artificial intelligence and other technological advances in the field. “Being a part of the puzzle around how we disseminate new lifesaving technologies is what really motivates us and gets us excited.”

Mentioned in this episode: https://mrionline.com/

If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Next Episode

The Power of a Rare Disease Community - Luke Rosen, Founder of KIF1A.org

“You know, it's easy to say that default answer that everything's okay, but it's really not. She's lost a lot of her vision, she's got hundreds of seizures at night, and she's having difficulty walking,” shares Luke Rosen about his eight-year-old daughter Susannah. She was born with KIF1A-associated neurological disorder -- or KAND -- a rare, degenerative genetic disease for which there is currently no cure or treatment. On this episode of Raise the Line, Luke talks about how he and his wife Sally summoned the strength to move beyond their family’s own challenges to create KIF1A.org which is working to rapidly discover a treatment for all patients and families affected by this devastating disorder, but to also create a supportive community. “Five years later, we have approximately four hundred families around the world that we've identified and there's not one family I know that doesn't play a significant role in what we do.” Thanks to that global community and partnerships with the Chan Zuckerberg Initiative, Columbia University, the n-Lorem Foundation, the Jackson Laboratory and many other organizations, there’s reason to be hopeful, as Luke shares with host Shiv Gaglani. “Susannah has been fortunate enough to just have started an experimental treatment. We really are on the brink of several things for, hopefully, the entire community.” Tune in for a candid and moving look at how families and supportive scientists and healthcare providers are mobilizing to fight back against a rare and pernicious threat to their children.

Mentioned in this episode: https://www.kif1a.org/

If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast