Mezzion Pharmaceuticals Announces Dr. Rahul Rathod of Boston Children's Hospital and Harvard Medical School as the Global Principal Investigator for the Confirmatory Pivotal Phase 3 Trial FUEL-2
FORT LEE, N.J., March 1, 2024 /PRNewswire/ -- Mezzion Pharmaceuticals is a rare disease company conducting the confirmatory pivotal phase 3 clinical trial in Fontan subjects: The Fontan Udenafil Exercise Longitudinal Assessment Trial- 2, or FUEL-2 trial. The company is pleased to announce... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 1, 2024 Category: Pharmaceuticals Tags: TRI Source Type: news
Harry Sammut's family face a crippling bill to get him to the US for treatment not available in Australia for his rare disease - as CEO says kids regularly die here 'because of paperwork'
The mother of a young boy battling a rare disease has revealed her family are set to spend more than $130,000 so her son can receive potentially life-saving treatment in America. (Source: the Mail online | Health)
Source: the Mail online | Health - March 1, 2024 Category: Consumer Health News Source Type: news
Chiesi Global Rare Diseases Launches " Rethink Acromegaly " Campaign to Provide Important Disease Education and Support to the Acromegaly Community
Rethink Acromegaly is created by Chiesi and members of the acromegaly community to help people living with the condition, along with their caregivers and families, to make informed healthcare decisions Program includes resources for healthcare professionals to optimize disease management to…#rethinkacromegaly #chiesi #boston #chiesigroup #rethinkacromegalycom #acromegaly #giacomochiesi #jillsisco #acromegalycommunity #globalrarediseases (Source: Reuters: Health)
Source: Reuters: Health - March 1, 2024 Category: Consumer Health News Source Type: news
FDA Helps to Deliver Meaningful Advances for Rare Disease Patients
We remain committed to supporting rare disease research, engaging patients and caregivers to understand their unique perspectives, and enhancing our review processes to advance medical products for rare disease patients. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - February 29, 2024 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Natural History Studies and Registries in the Development of Rare Disease Treatments - 05/13/2024
Natural History Studies and Registries in the Development of Rare Disease Treatments (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - February 29, 2024 Category: Drugs & Pharmacology Authors: FDA Source Type: news
England Rare Diseases Action Plan 2024: main report
This report details progress against 29 existing actions as well as introducing 7 new actions.Policy paperDepartment of Health and Social Care - publications (Source: The Kings Fund - Health Management Specialist Collection)
Source: The Kings Fund - Health Management Specialist Collection - February 29, 2024 Category: Health Management Authors: The King ' s Fund Library Tags: Quality of care and clinical outcomes Source Type: news
Rare Disease Day 2024: IndoUSrare's Frontline Efforts in Tackling Global Inequities
Indo US Organization for Rare Diseases (IndoUSrare) has always been a steadfast ally to rare disease patients, and Rare Disease Day 2024 will be no exception. Beyond honoring these individuals on February 29 through online and in-person events, IndoUSrare will be addressing the enduring... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 29, 2024 Category: Pharmaceuticals Tags: TRI Source Type: news
Gene therapy company launched by parents, Nobel Prize winner doses 1st patient in ultra-rare disease
Children in the study will be followed for five years. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 28, 2024 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
Advancing rare disease breakthroughs with genomics, AI and innovation
From the forefront of medical research to the front lines of patient care, scientists and clinicians at the Mayo Clinic Center for Individualized Medicine are making notable progress in rare disease diagnostics and treatments. Patient by patient, they are dedicated to bringing hope, answers and healing to those who have tirelessly searched for solutions to their conditions. The pioneers in the field, including Margot Cousin, Ph.D., and Filippo Pinto e Vairo, M.D., Ph.D., are harnessing… (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - February 28, 2024 Category: Databases & Libraries Source Type: news
InnovationRx: Blueprint Says Its Rare Disease Drug Could Be A Blockbuster
InnovationRx is your weekly digest of healthcare news. Sign up! (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - February 22, 2024 Category: Pharmaceuticals Authors: Katie Jennings, Forbes Staff Tags: Science /science Innovation /innovation Healthcare /healthcare Editors' Pick editors-pick Newsletter No Paywall no-paywall technology alexknappblog premium Source Type: news
Down syndrome identified in 2600-year-old infants through their DNA
Between 770 and 550 B.C.E., in a hilltop village called Alto de la Cruz near the modern Spanish city of Navarro, one infant girl received an unusual burial: Although people in Iron Age Spain usually cremated their dead, the girl was laid to rest beneath the floors of an elaborately decorated dwelling, with grave goods including bronze rings, a shell from the Mediterranean, and three whole sheep or goats.
And she wasn’t alone: Archaeologists working in the 1940s and ’50s found dozens of infants buried below the floors in Alto de la Cruz and another nearby village built more than 2600 years ago. “The main questio...
Source: ScienceNOW - February 20, 2024 Category: Science Source Type: news
PCORI Advisory Panels: Panel Openings
Patient-Centered Outcomes Research Institute (PCORI) is accepting nominations and applications for 5 advisory panels: Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Healthcare Delivery and Disparities Research, Advisory Panel on Patient Engagement, Advisory Panel on Clinical Trials, and Advisory Panel on Rare Disease. Offers overview of requirements for panelists, the application process, and a link to the nomination and application webpage. Nominations are due March 29, 2024. (Source: News stories via the Rural Assistance Center)
Source: News stories via the Rural Assistance Center - February 15, 2024 Category: Rural Health Source Type: news
A Man Has Died From Alaskapox. Here ’s What We Know About the Virus
Alaska’s health department reports that the first person in the state has died from a recently discovered virus called Alaskapox.
The elderly man—who was immunocompromised due to cancer treatments—first noticed an unusual lesion in his right armpit last September, according to Alaska health officials who spoke to TIME about the case. He was prescribed antibiotics at his local emergency room on the Kenai Peninsula, but after multiple visits and a worsening, painful infection, he was transferred to a hospital in Anchorage.
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The patient tested positive for ...
Source: TIME: Health - February 13, 2024 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized healthscienceclimate Source Type: news
A Collaborative Approach to Advancing Access and Equity in Rare Disease Clinical Trials, Upcoming Webinar Hosted by Xtalks
In this free webinar, learn about the importance of creating patient-friendly clinical trials to study rare diseases. The featured speakers will discuss how to engage and support patients in rare disease clinical trials. The speakers will share lessons learned in a successful Pompe... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 13, 2024 Category: Pharmaceuticals Tags: FVT Source Type: news
A Gen Xer moved from California to Colombia after overcoming a rare disease. The cost of living is much cheaper, and life is more peaceful
Andy Wiesmann and his wife met in Colombia. Andy Wiesmann Andy Wiesmann moved from California to Mexico to Colombia in search of a cheaper cost of living. He couldn't afford housing in California after developing a rare autoimmune disorder. He said Colombia has perfect weather, much lower costs,…#andywiesmann #colombia #mexico #medellín #goldenstate #census #censusbureau #acs #californians #middleeast (Source: Reuters: Health)
Source: Reuters: Health - February 10, 2024 Category: Consumer Health News Source Type: news
