Sentynl Therapeutics, Inc.

As part of our #VoicesOfSentynl series, we’re spotlighting Jason Rexroad, Sentynl’s Senior Director of Quality.    In #RareDisease, quality work often happens behind the scenes, but it plays an important role in the work teams do every day. We’re proud to share Jason’s perspective on the responsibility that comes with helping ensure patients and families can count on that work. Thank you, Jason, for sharing your thoughts and for the care, rigor, and accountability you bring to your work at #Sentynl.    #Leadership

#Sentynl welcomes healthcare workers, advocates, and media to engage with and learn from the rare disease community about patient and family experiences.    A recent CNBC Cures story shared one family’s path to answers after their son showed symptoms they could not explain. What followed was a long search for clarity, including multiple diagnoses, and an initial result that still left many questions unresolved.    Only later, after tests were rerun in light of a newly identified genetic disorder, did the family finally receive a diagnosis.    Stories like this are an important reminder that for many families, the diagnostic odyssey is not only medical. It can also bring emotional, financial, and practical strain. As rare disease receives broader visibility, we are encouraged to see more of these experiences brought into the conversation.    To read Max’s story, and others, visit: https://lnkd.in/eNFiFjMn.

This week, members of the #Sentynl team attended Asembia’s #AXS26 Summit in Las Vegas, where much of the focus was on how specialty medicines reach patients and the systems that support access to care.   The event was a valuable opportunity to hear how others think about access, support and the work required to help treatments reach patients. Thank you to Mike Cvijanovich, Kyle Marquardt, and John Hormuth for attending and participating in important conversations about the future of rare disease care!   #RareDisease

We’re proud to introduce the Sentynl #LeadershipSeries, highlighting perspectives from our founder and CEO, Matt Heck.    This month, Matt reflects on an important reality in #RareDisease — developing therapies is only one part of the equation when it comes to patients' care journey. Timely diagnoses and access to care also shape what is possible for these patients and families.    Swipe for his full perspective!    #Sentynl

Today, on #NationalDoctorsDay, we recognize the physicians who dedicate their lives to finding answers for rare disease patients. For patients with ultra-rare conditions, a doctor is more than a provider… they are often the first person to offer a name for previously unknown conditions and offer a path forward. Thank you to the clinicians who ask the hard questions and advocate for rare disease patients. You are #HealthcareHeroes and your curiosity and care change lives.

For many parents and caregivers in the rare disease community, the road to a diagnosis starts with uncertainty and is often paved with unanswered questions. This journey, the "diagnostic odyssey,” can take years, leaving families feeling isolated. Shortening this odyssey requires more than resilience: it requires early action. Prioritizing rapid whole genome sequencing helps identify a diagnosis early, which can guide the best course of action sooner, including any available treatments or clinical trials for eligible patients. At Sentynl, we honor the resilience of caregivers who refuse to stop searching for answers.

Today, we are proud to share that Sentynl has entered into an agreement with PRG S&Tech Inc. to acquire full rights to its investigational drug candidate, Progerinin (SLC-D011), for Hutchinson-Gilford Progeria Syndrome (HGPS). HGPS is a devastating disease, and we are committed to developing new treatment options for patients and their families. Progerinin has received orphan drug designation by the U.S. FDA, and we look forward to advancing the clinical development of this investigational therapy. Progerinin is not currently approved by FDA or any other health authority. Read more in our press release here: https://lnkd.in/emUmJH35 #RareDisease #Progeria

Thank you to PharmaBoardroom for the feature interview with our CEO Matt Heck. We appreciate the opportunity to share more about Sentynl and our commitment to advancing therapies for patients with rare diseases.

Navigating the global landscape of rare disease takes a team that thrives on collaboration. For Kyle Marquardt, Senior Manager of Commercial Operations at Sentynl, the strength and expertise of the colleagues around him are vital elements in navigating industry complexity and meeting patient needs around the world. #Sentynl is a special place to work because of leaders like Kyle and a team that consistently rises to the challenge to ensure we can reach the patients who are counting on us! #VoicesOfSentynl #RareDisease

Seconds count when it comes to a baby’s developing brain. This is especially true with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare genetic condition that prevents the body from breaking down toxic sulfites. Without treatment, MoCD Type A causes rapid, irreversible neurological damage. Because symptoms often include seizures and feeding difficulties, MoCD Type A is particularly challenging to diagnose. This "diagnostic odyssey" costs precious time, but greater awareness of symptoms and testing options can change outcomes. Rapid whole genome sequencing is critical to helping patients and families identify a diagnosis and pursue appropriate treatment options. Knowledge is the first step toward hope. Learn more about the signs of MoCD Type A at the link: https://lnkd.in/e-YPDSWK. #MoCDTypeA #RareDisease #GeneticTesting