Cumulative Trauma Disorders

KIBBUTZ REIM, Israel, March 13, 2024 /PRNewswire/ -- In a significant advancement for mental health care, Madrigal Mental Care is excited to announce the successful completion of its pivotal nanotechnology research on the use of Ketamine as a treatment for trauma related disorders such as Post-Traumatic Stress Disorder (PTSD) and depression. This groundbreaking study marks the beginning of a new chapter, as Madrigal gears up to initiate in-human clinical trials. The research underscores the potential of Ketamine combined with Madrigal's proprietary nanotechnology to offer a transformative approach to PTSD treatment, aiming to address the core of the trauma rather than merely managing symptoms. The global surge in PTSD, highlighted by the recent conflict in Israel following the October 7th attacks, underscores the urgency of addressing this epidemic. Madrigal, originating from Kibbutz Re'im in Israel's south, has been dedicated to combating PTSD, a commitment that has intensified in the wake of these events. The company is at the forefront of developing innovative treatments to meet the growing need for effective PTSD interventions, particularly in regions hardest hit by conflict. "As the company's CEO, and as a survivor of the events of the seventh of October, I am personally committed to leveraging Madrigal's unique and groundbreaking technology to address the severe post-trauma challenges we've identified." said David Gabay, CEO of Madrigal, "Our goal is to begin human trials this year and expedite FDA approval processes, aiming to provide a revolutionary treatment by 2026. The scale of mental health issues emerging necessitates immediate and bold action, and the support from our international community is critical for turning our vision into reality." Dr. Yuval Neria, professor at Columbia University Irving Medical Center, specializing in trauma and PTSD and clinical advisor to Madrigal, has been at the forefront of documenting this crisis. Collaborating with Israeli scientists, their recent paper, "PTSD, depression, and anxiety after the October 7, 2023 attack in Israel: a nationwide prospective study" provides a detailed report on the magnitude of the mental health crisis in the aftermath of the October 7th attacks in Israel. Dr. Neria's work not only sheds light on the scale of the issue but also emphasizes the urgent need to develop innovative treatments that can effectively address the complex nature of trauma related disorders including PTSD and depression. "Based on our own studies as well as on other emerging reports, I am deeply concerned that without effective, innovative treatments, numerous civilians and soldiers will suffer from chronic, debilitating mental health problems in the wake of the October 7th terrorist attack and the following war" said Prof. Neria, highlighting the need to quickly test the efficacy of Madrigal's technology in humans with trauma related disorders including PTSD and depression. As Madrigal prepares to embark on the next phase of clinical trials, the company extends an open invitation to individuals and organizations interested in learning more or collaborating on this journey. With a commitment to redefining mental health treatment, Madrigal is poised to make a significant impact on the lives of those affected by PTSD. For more information or to discuss potential collaborations, please reach out to Madrigal's press office. Press contact : Yael Ayalon CMO E: [email protected] T: +972.54.947.4380 W: Logo - SOURCE Madrigal Mental Care

In the driver’s seat on Alnylam’s cross-country road trip is Bernice Bennett, an author and genealogist. Mapped out along her route are stops to talk with a doctor who’s an expert on hATTR amyloidosis, as well as families that have been affected by the disease. A non-exhaustive list of supplies needed for a successful family road trip: a full tank of gas, plenty of snacks, a meticulously curated playlist and, according to Alnylam Pharmaceuticals, a thorough conversation about your family’s health history. The pharma has launched a new campaign, “Family Health History Road Trip,” to encourage people to have those conversations with their relatives, in hopes of identifying hereditary diseases they might be carrying as early as possible. The campaign is focused specifically on hereditary ATTR (hATTR) amyloidosis, a rare inherited condition in which a genetic variant leads to misfolded TTR proteins, which can then build up in the heart and other organs and tissues. Diagnosis of the rapidly progressing disease can take several years after symptoms begin, since those symptoms—which include a range of reactions from carpal tunnel syndrome to dizziness and shortness of breath to unintentional weight loss—overlap with those of many other, less-rare conditions. Currently, according to the American Heart Association, people with hATTR amyloidosis typically have a life expectancy of between 2.5 and 5 years after they’re diagnosed. One participant in Alnylam’s campaign said in a company release Wednesday that it took appointments with nine doctors over the course of seven years for her to finally receive a definitive diagnosis. “Once I mentioned my uncle’s history of amyloid neuropathy to my doctor, I was immediately offered a genetic test and finally received a diagnosis,” she said. “My symptoms were so different from my uncle’s that I never thought to discuss his health history with my doctor when I started my search for answers. If I had, I could have potentially been diagnosed and started on a disease management plan much earlier.” In the driver’s seat on Alnylam’s cross-country road trip is Bernice Bennett, an author and genealogist. Mapped out along her route are stops to talk with a doctor who’s an expert on hATTR amyloidosis, as well as families that have been affected by the disease. A highlight video from the trip shows Bennett going through family photo albums and chatting with patients’ family members about the process of uncovering the diagnosis behind what seemed like “a mystery illness.” She also meets with a doctor, who notes that “delay in diagnosis in a person is not only resulting in delay in diagnosis in them but also delay in diagnosis in the subsequent generations of their families.” “When we speak about family history, we often focus on the stories about what country we came from, where family immigrated to, or what types of jobs or professions they held; what we don’t often focus on is the health history of those who have walked before us,” Bennett says in the video. The highlight reel also gives a preview of Bennett’s route, starting in Chicago and winding through the Midwest before heading east for Massachusetts. And while the Family Health History Road Trip doesn’t overtly tout any branded treatments for the disease, the “treatment options” tab on the campaign’s accompanying website does redirect visitors to the site for Amvuttra, an RNAi drug from Alnylam that’s indicated to treat nerve damage in hATTR amyloidosis patients. The road trip-themed campaign is the latest in a string of hATTR amyloidosis programs that Alnylam has launched in recent years. Back in 2018, its “Bridge the Gap” campaign spotlighted a single extended family with cases of the disease spanning multiple generations, while another effort that launched last year focused on raising awareness of the rare disease in Europe via animated patient testimonial videos after Amvuttra was cleared on the continent. “Alnylam has been learning from the hATTR amyloidosis community for nearly two decades, and during that time, I’ve witnessed firsthand the devastating impact a delayed diagnosis can have on an individual’s quality of life and on their entire family,” Tiffany Patrick, VP of patient advocacy and engagement at Alnylam, said in this week’s announcement. “The Family Health History Road Trip program underscores Alnylam’s commitment to supporting improved health outcomes for the hATTR amyloidosis community.”

CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the launch of the Family Health History Road Trip , its program designed to encourage conversations between family members about their health history so they can discuss with their doctor to better understand their risk for developing an inherited disease like hereditary ATTR (hATTR) amyloidosis. Inspired by the traditional family road trip, the program follows genealogist and health educator Bernice Bennett as she embarks on a cross-country road trip to meet with a physician expert in hATTR amyloidosis and affected families to learn more about the disease, hear patients’ journeys to diagnosis and explore how knowledge of family health history can drive an earlier diagnosis. “When we speak about family history, we often focus on the stories about what country we came from, where family immigrated to, or what types of jobs or professions they held; what we don’t tend to focus on is the health history of those who have walked before us,” said Bennett. “Family history is considered one of the most important risk factors for health problems. Knowing your family’s health history empowers you with important information to share with your doctor when seeking a diagnosis or determining your risk for developing an inherited disease like hATTR amyloidosis.” hATTR amyloidosis is a rapidly progressive disease caused by an inherited variant, or change, in the transthyretin (TTR) gene, which causes the TTR protein to take on an abnormal shape, misfold and collect as amyloid deposits throughout the body, including the nerves, heart and digestive system, resulting in symptoms of the disease. Common symptoms may include carpal tunnel syndrome, tingling/numbness in the hands or feet, weakness, shortness of breath, diarrhea, constipation, dizziness and unintentional weight loss. These symptoms are similar to those of more common conditions and can vary widely among people with the condition, even within families, which may lead to misdiagnoses or delays in receiving an accurate diagnosis. In fact, patients may not receive a correct diagnosis until 3 to 6 years after symptom onset, resulting in significant disease progression and impacting entire families who remain unaware that there is an inherited disease in the family. “It took seeing nine doctors over seven years for me to finally receive a diagnosis of hATTR amyloidosis,” said Christine, a Family Health History Road Trip participant who is living with hATTR amyloidosis. “Once I mentioned my uncle’s history of amyloid neuropathy to my doctor, I was immediately offered a genetic test and finally received a diagnosis. My symptoms were so different from my uncle’s that I never thought to discuss his health history with my doctor when I started my search for answers. If I had, I could have potentially been diagnosed and started on a disease management plan much earlier.” With an inherited disease like hATTR amyloidosis that progresses rapidly, early diagnosis is crucial. When a diagnosis is delayed, appropriate management of the disease is also delayed, which is especially problematic as symptoms of hATTR amyloidosis can worsen over time and cause irreversible damage. That is why having health history conversations within families is so important – it equips current and future generations with important health information to share with their doctor and take action. A genetic test can determine whether a person carries one of the more than 120 variants in the TTR gene associated with hATTR amyloidosis. Once a gene variant is identified, family members of an affected individual can share this information with their doctor to help determine their own risk for the disease. “Alnylam has been learning from the hATTR amyloidosis community for nearly two decades, and during that time, I’ve witnessed firsthand the devastating impact a delayed diagnosis can have on an individual’s quality of life and on their entire family,” said Tiffany Patrick, MPH, MBA, Vice President, Patient Advocacy and Engagement at Alnylam Pharmaceuticals. “The Family Health History Road Trip program underscores Alnylam’s commitment to supporting improved health outcomes for the hATTR amyloidosis community.” hATTR amyloidosis is an autosomal dominant condition – meaning each child of one parent with hATTR amyloidosis has a 50 percent chance of inheriting the genetic variant that causes the condition. While a family member may inherit the TTR gene variant, having the variant does not necessarily mean that they will develop the disease. Although anyone can be at risk for developing hATTR amyloidosis, it is more common in certain ethnicities, such as people of African, Brazilian, French, Irish, Japanese, Portuguese and Swedish descent. To follow along on Alnylam’s Family Health History Road Trip and learn more about hATTR amyloidosis, the role of genetic testing, as well as ways you can start discussing family health history with loved ones, visit hATTRbridge.com/roadtrip . About Alnylam Pharmaceuticals Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO ® (patisiran), AMVUTTRA ® (vutrisiran), GIVLAARI ® (givosiran), OXLUMO ® (lumasiran), and Leqvio ® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P 5 x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam , or on LinkedIn , Facebook , or Instagram .