rare disease day

In this episode, Ayesha talked about Rare Disease Day 2024, a global movement held on the last day of February each year that focuses on raising awareness about rare diseases including their impact on patients’ lives. This year, Rare Disease Day falls on February 29, the rarest day in a leap year, making it even more special.

Ayesha spoke about how the Rare Disease Day campaign helps increase awareness about rare diseases, promotes research and development, supports advocacy for policies supporting patients and their families and brings together a global rare disease community.

Advancements in rare disease research are leading to innovative new treatments for various rare conditions, including genetic disorders, which make up just over 70 percent of all rare diseases.

Hear more about new rare disease drugs approved by the FDA in the past couple of years. Also hear about some of the latest trends in rare disease research, including innovative trial designs, patient-centric approaches, the use of cutting-edge technologies like AI and international collaborations.

Read the full articles here:

Rare Disease Day 2024: Highlighting Statistics and Patient Stories
New Rare Disease Drugs and Research Advancements

For more life science and medical device content, visit the Xtalks Vitals homepage.

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On this special episode of the Pharma Podcast, we're honouring Rare Disease Month this February by delving into the personal and profound stories from the front lines of rare diseases in Canada. Our focus is on the unique challenges, healthcare experiences, and the resilience required to navigate life with a rare condition. In celebration of this important month, I’m thrilled to have Dr. Sonia Rehal with us. Sonia is not only courageously living with a rare disease but also actively participating in raising awareness and advocating for better support and research for rare diseases in Canada.

Sonia's journey offers us a window into the day-to-day realities faced by individuals with rare diseases, from the quest for diagnosis to access to specialised care, and underscores the importance of community support. Her insights are invaluable in dispelling misconceptions about rare diseases and highlighting the ongoing efforts to improve the lives of those affected.

Join us as we explore Sonia's story, the broader landscape of rare diseases in Canada, and what hope and advancements lie on the horizon for patients and their families. 

Through this episode, we hope to convey a powerful message: while each rare disease may affect a small number of individuals, collectively, these conditions are not that rare at all. 

Lipodystrophy Canada Website:

https://lipodystrophy-canada-foundation.wiredwebsites.org/

Facebook Page:

https://www.facebook.com/profile.php?id=100092509705779

Our Email address: 

lipodystrophycanada@gmail.com

On this episode of the PharmaPodcast, I have an extra special guest. Artem Brytchenko is CEO at Platforce, a company based in Ukraine that he continues to run, in spite of all the adversity he is facing during a time of war.  I’m sure you will find this interview not just informative, but also Inspirational.

Contact Information:

Artem Brytchenko 

Chief Executive Officer | Platforce

E:  support@platforce.io

W: https://platforce.io/

On this episode of the Pharma Podcast, my extra-special guest is Dr. Durhane Wong-Rieger,  President & CEO at Canadian Organization for Rare Disorders.

To mark Rare Disease Day, which we celebrate on the last day of February, I can think of no better guest than Dr. Durhane Wong-Rieger to bring awareness to Rare Disease Day.

She also gives a great overview of CORD and its mission.

To learn more about CORD, upcoming events, or to make a donation, please visit: www.raredisorders.ca

You can also follow CORD on social media:

https://www.facebook.com/RareDisorders

https://twitter.com/raredisorders

https://www.youtube.com/user/CORDRareDisorders





 

In this episode, the editorial team discussed Rare Disease Day which is celebrated every year on February 28 (or February 29 in leap years). This year’s theme was “Share Your Colours,” where individuals with rare diseases were encouraged to share their experiences and aspirations to help raise awareness. The campaign from rarediseaseday.org included a touching video in which patients with rare diseases shared their stories. The editorial team discussed the encouraging growth seen in recent years in rare disease clinical research in the life science industries, providing hope for improved diagnosis, care and treatments for patients with rare diseases.

To mark the end of Black History Month, the team also talked about the underrepresentation of Black people in clinical trials. Although Black people are disproportionately affected by certain diseases due to a combination of socioeconomic and genetic factors, their rates of participation in clinical trials remain concerningly low. In the US, Black people only constitute five percent of all clinical trial participants. Studies show historic medical mistrust among Black people for healthcare systems is the major barrier to trial participation, along with socioeconomic barriers and a lack of patient education. Community outreach and active engagement by the medical and scientific communities are key to improving clinical trial participation among Black people.

This week’s Xtalks Life Science Podcast was sponsored by Elligo Health Research.

Read the full articles here: 

Sharing Colours on Rare Disease Day 2022

Why Black People Remain Underrepresented in Clinical Trials

For more life science and medical device content, visit the Xtalks Vitals homepage.

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Twitter: @Xtalks
Instagram: @Xtalks
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YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a degenerative neurological disease most often associated with older while males. However, it is not exclusive to them, and obtaining a diagnosis as a young woman can be tricky. The women of Her ALS story are patients and spokespeople who are trying to change that by sharing their stories with the world. Listen now to hear their stories, and learn more at heralsstory.org.

By definition, a rare disease is one that only affects a handful of people in the world. But with more than 300 million people collectively affected by these conditions and only few approved treatments available, rare diseases constitute a major unmet medical need. Christina Ström Möller from Swedish biopharmaceutical company Sobi walks us through the scientific, commercial and human challenges of the rare disease landscape.

Tune in to find out:

• What drug development, testing and distribution look like when patients are few and far apart 
• Why patient narratives are especially important when monitoring the safety of rare disease drugs
• How patient organisations work to support rare disease patients and raise awareness of their condition

Want to know more?

Read the stories of patients living with rare diseases on the Sobi website.

Show your support for Rare Disease Day on 28 February by sharing campaign materials online with the hashtag #RareDiseaseDay or joining one of the many events planned around the world.

Rare Disease Day is organised by EURORDIS-Rare Diseases Europe, a non-profit alliance of rare disease patient organisations from 73 countries.

When assessing the safety of rare disease medicines, also known as orphan drugs, it is important to include all available sources of data.

A recent review of orphan drug policies found that several low- and middle-income countries had developed such policies in the last decade ­– although disparities between countries with different income levels still remain.

A survey conducted by EURORDIS in 2020 found that the COVID-19 pandemic exacerbated the many challenges that people living with a rare disease face.

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