Hosted on MSN11h
Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing ...
STAT10d
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?
Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...
Opinion: California’s Rare Disease Council Will Transform Care for People with ALS
California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
Native News Online4dOpinion
Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Guest Opinion. About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare ...
Minnesota Daily4d
Parents of children with rare diseases share their stories for Rare Disease Day
explained how patients with rare diseases can utilize telehealth services nationally and internationally. The University houses the Rare Disease Advisory Council (RDAC), a support network for parents ...
YTN22h
Establishment of a basis for supporting the production and seller of medical devices for rare diseases
The basis for support has been established for those who produce and sell medical devices and patient special foods used to ...
Cannabinoid hyperemesis syndrome: a rare but troubling health risk for cannabis users
A rare but severe condition, cannabinoid hyperemesis syndrome is caused by heavy cannabis use and is "marked by nausea, ...
GEN20h
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
Siegel Rare Neuroimmune Association Hosts 2024RNDS
Sessions that focused on diagnosis and acute treatment of rare neuroimmune disorders (RNDs). A panel of people with these ...
health.economictimes.indiatimes9d
Patents and high price barriers: Patients with rare diseases face woes to access medicines from MNCs
Patients with rare diseases in India, like Saifullah Khalidi with spinal muscular atrophy (SMA), struggle to afford ...
Yahoo Finance9d
Agios to Present New Data on Mitapivat and Tebapivat in Rare Blood Disorders at 66th ASH Annual Meeting and Exposition
Nov. 05, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases ...
Tahlequah Daily Press7d
CHIEF CHAT: Cherokee Nation rare disease summit to address overlooked health care needs
About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare Disease Summit ...