We are excited to announce that we have entered into an agreement to be acquired by Alexion in a transaction that we hope will accelerate our mission to deliver genetic medicine to patients with rare diseases. Learn more in the press release issued today https://t.co/hpMGmhiG0b
We have had the opportunity to connect with many individuals whose journeys with #organicacidemias, including #methylmalonicacidemia, represent the strength and resilience that characterize the #raredisease community. Hear their stories: https://t.co/wMhsXQ9YdO
Looking forward to the unveiling of the @OrganicAcidemia Association’s new website! On its current site, the OAA shares a range of resources to help educate and support the #organicacidemia community. Take a look: https://t.co/eKzwcTZHqh
#ICYMI Watch a recording of the @OrganicAcidemia Association’s Family Conference, featuring presentations from leaders in research and patient care: https://t.co/TInwNQXe6K
The National Organization for Rare Disorders’ extensive Rare Disease Database provides important information to support individuals and caregivers affected by specific #rarediseases. Check it out here: https://t.co/sXSlwJnT4H @RareDiseases
On Thursday, Aug. 18 at 3:10pm ET, Nick DiGioia, Manager, Process Development, will deliver a presentation titled “Optimization of an AAV Purification Process to Accommodate Increased Upstream Yield and Reduce Manufacturing Bottlenecks.”
Members of our CMC team are presenting at the #CHIBioprocessingSummit in Boston this week! Check out the full program agenda here: https://t.co/CNnCWv5O4N @CHI_Healthtech
Lauren M. Drouin, PhD, Director, Analytical Development, led a presentation titled “Development of a Potency Assay Matrix for the Characterization of AAV Gene Editing Products,” and joined several #biotech colleagues for the “CMC Strategies for Gene Therapies” panel discussion.
In #MethylmalonicAcidemia, toxic metabolites accumulate in the liver, muscle tissue and brain, leading to potential symptoms such as developmental delays, organ damage and other health challenges. Learn more about the impact of MMA: https://t.co/MTBKR8ljUM
This month, the @OrganicAcidemia Association is putting a spotlight on members of the #methylmalonic acidemia community in its August OAA Calendar. Take a look:
Genetic testing is an important tool used by many #genetic counselors to support the diagnosis of a range of #rarediseases. Visit @GlobalGenes’ resource center to learn more about the advantages of #genetictesting: https://t.co/rUjWcor5Le
To learn more about our research in #rarediseases affecting individuals from infancy through adulthood, check out these publications: https://t.co/iFrSDjsj6R
Thank you @OrganicAcidemia for the opportunity to participate in this year’s Family Conference! We know the important discussions that took place throughout the meeting will play a key role in expanding support for members of the #organicacidemia community.
“The love we have for our child is amazing and makes all of the challenges worth it. We’re so glad that she is here with us.”
In this article, Carina and Luke Farkas share the story of their family’s journey with #methylmalonicacidemia: https://t.co/Z7XcYHZ0dc
At the @OrganicAcidemia Association’s Family Conference, we had the privilege to connect in-person with the #methylmalonicacidemia community & hear impactful stories from families navigating the challenges of MMA with resilience. Thank you to the OAA for this special opportunity.
Today we join our #biotech colleagues in recognizing the important work that lies ahead to advance #healthequity and create a more equitable, just society. Learn about #Juneteenth: https://t.co/Iv2lLyQo5R
Dr. Mariana Nacht, Chief Scientific Officer, discusses our approach to #geneediting in this Drug Development & Delivery article: https://t.co/yQK94tKbGW