Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Akari Therapeutics files a new patent for AKTX-102, an antibody-drug conjugate targeting CEACAM5, enhancing its intellectual property portfolio. This move strengthens its pipeline as the company continues to develop innovative therapies.
Erasmus Medical Center is recruiting participants for a natural history study on CAMK2-related synapthopathies, focusing on the role of the CAMK2 gene. This study aims to gather data on the progression and characteristics of these rare conditions.
The MIHRA initiative focuses on gathering patient-rooted insights to shape research in myositis and related conditions. Sponsored by multiple organizations, including Myositis International and The Myositis Association, this project emphasizes qualitative investigations to better understand patient experiences.
The CDC highlights areas at risk for chikungunya transmission, advising U.S. travelers to consider vaccination. This guidance reflects ongoing public health efforts to manage and prevent outbreaks.
The CDC introduces the DOSE-SYS Dashboard to enhance nonfatal overdose syndromic surveillance using electronic health record data. This initiative aims to improve public health responses to overdose incidents.
The CDC has updated its Infant Mortality Dashboard, providing quarterly estimates of infant, neonatal, and postnatal deaths along with death rates for the five leading causes of death. This tool aims to enhance public health policy and inform stakeholders about critical mortality trends.
The CDC launches the STOP Program to recruit and train international public health consultants, aiming to enhance global health initiatives. This program reflects a strategic move to bolster public health expertise worldwide.
The CDC has released quarterly provisional estimates of infant mortality for Q2 2023, providing critical data for health policy discussions. These estimates are essential for understanding trends and informing interventions aimed at reducing infant mortality rates.
HHS updates civil monetary penalty amounts to reflect inflation adjustments as mandated by the Federal Civil Penalties Inflation Adjustment Act Improvements Act of 2015. This regulatory change may impact compliance costs for healthcare organizations.
NORD's virtual event will update attendees on key legislative issues affecting rare disease advocacy in 2026. The session aims to engage participants in ongoing advocacy efforts and highlight opportunities for involvement.
The Administration for Children and Families seeks to extend approval for Reviewer Recruitment Forms under OMB #0970-0477, with updated burden estimates but no changes to the terms. This proposal is part of ongoing efforts to streamline administrative processes.
Rare Disease Day, observed on February 28, aims to raise awareness and advocate for equity in healthcare for individuals with rare diseases. Coordinated by EURORDIS and over 70 patient organizations, the campaign highlights the importance of community voices in driving change.
Global Genes announces the launch of RARE-X, a patient-driven initiative aimed at collecting symptom and patient-reported outcome data for rare diseases. This effort seeks to enhance research and understanding of rare conditions through direct patient engagement.
A new NIH-funded study establishes a clear link between severe chronic traumatic encephalopathy (CTE) and increased dementia risk. This research could influence future therapeutic strategies and patient management for individuals with a history of head trauma.
The CDC has launched a new resource page dedicated to providing information for patients and caregivers about preventing falls among older adults. This initiative aims to enhance awareness and support for a vulnerable population.
The CDC has released new guidelines addressing obesity and overweight, emphasizing the need for comprehensive strategies to combat these conditions. The guidelines aim to inform policy decisions and public health initiatives.
The CDC provides updated information on rabies, detailing its transmission, at-risk populations, and prevention strategies. This resource aims to enhance public awareness and safety regarding this preventable disease.
The CDC emphasizes summer swim safety, providing guidelines to prevent accidents and ensure safe swimming practices. This initiative aims to raise awareness among communities about water safety.
The U.S. Department of Health and Human Services rescinded guidance aimed at ensuring nondiscriminatory access to health care at pharmacies. This decision may impact how pharmacies comply with federal civil rights laws regarding access to reproductive health care services.
The FDA has determined the regulatory review period for REZDIFFRA, which is necessary for the patent extension application process. This determination is part of the legal requirements for patent claims related to human drug products.
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