5 health bills impacting California patients in 2025California's new health-related laws aim to enhance access and reduce financial barriers for patients with chronic and rare diseases starting January 1, 2025.
For kids with rare genetic disorders, customized CRISPR treatments offer hopeLucy Landman suffers from a rare genetic disorder with no current treatment; CRISPR shows promise, yet funding remains a critical hurdle for research.
Why Early Diagnosis of PAH is So ImportantIncreasing awareness of pulmonary arterial hypertension (PAH) is crucial for early diagnosis and better patient management.
For kids with rare genetic disorders, customized CRISPR treatments offer hopeLucy Landman suffers from a rare genetic disorder with no current treatment; CRISPR shows promise, yet funding remains a critical hurdle for research.
Why Early Diagnosis of PAH is So ImportantIncreasing awareness of pulmonary arterial hypertension (PAH) is crucial for early diagnosis and better patient management.
Toronto couple fundraising for Boston lab they hope can help their daughter | CBC NewsA Toronto family is fundraising for research on Selenon Related Myopathy to develop treatment for their daughter's ultra-rare disease.
Can AI shake-up translational research?AI-driven tools like the Biomedical Data Translator can expedite treatment for rare diseases, offering hope in translational medicine.
Using AI to repurpose existing drugs for treatment of rare diseases - Harvard GazetteAn AI tool called TxGNN can identify drug candidates for over 17,000 rare diseases, potentially addressing many previously untreated conditions.
Can AI shake-up translational research?AI-driven tools like the Biomedical Data Translator can expedite treatment for rare diseases, offering hope in translational medicine.
Using AI to repurpose existing drugs for treatment of rare diseases - Harvard GazetteAn AI tool called TxGNN can identify drug candidates for over 17,000 rare diseases, potentially addressing many previously untreated conditions.
Exploring the Elusive Genetic Causes of Rare Autoimmune Disease - News CenterThe study reveals new genetic insights into APECED, identifying non-coding mutations that impair the AIRE gene's function in patients lacking typical variants.
Influencer opens up about living with 'extremely rare eye condition'Gerry, a beauty influencer, has megalocornea, a rare condition that causes large eyes, emphasizing their natural appearance over cosmetic enhancements.
A disease that makes children age rapidly gets closer to a cureGene editing may offer hope for treating ultrarare conditions like progeria, potentially paving the way for treatments for other genetic diseases.
A Disease That Makes Children Age Rapidly Gets Closer to a CureA cure for progeria through gene editing may be imminent.
A disease that makes children age rapidly gets closer to a cureGene editing may offer hope for treating ultrarare conditions like progeria, potentially paving the way for treatments for other genetic diseases.
A Disease That Makes Children Age Rapidly Gets Closer to a CureA cure for progeria through gene editing may be imminent.
Imagine getting life-saving drugs to sick people without relying on big pharma? We may have found a way | Dr Catriona CrombieHealthcare providers are finding innovative ways to make life-saving treatments more accessible for patients with rare diseases.
AI Is Transforming Drug Matching for Cancer, Rare Diseases | EntrepreneurAI tools are revolutionizing drug discovery for rare diseases by repurposing existing medications, leading to cost-effective and efficient screening processes.
CDC alerts doctors to watch for rare, serious bacterial infection appearing with unusual symptomsCases of rare, serious meningococcal infections are rising in the US, affecting middle-aged adults.A certain strain of Neisseria meningitidis bacteria is causing these unusual infections with higher fatality rates than typical cases.
CDC alerts doctors to watch for rare, serious bacterial infection appearing with unusual symptomsRare meningococcal infections are on the rise in the US with higher fatality rates, affecting middle-aged adults.Infections caused by strain ST-1466 in the Y subgroup are prevalent, spreading through respiratory secretions or close contact.
CDC alerts doctors to watch for rare, serious bacterial infection appearing with unusual symptomsCases of rare, serious meningococcal infections are rising in the US, affecting middle-aged adults.A certain strain of Neisseria meningitidis bacteria is causing these unusual infections with higher fatality rates than typical cases.
CDC alerts doctors to watch for rare, serious bacterial infection appearing with unusual symptomsRare meningococcal infections are on the rise in the US with higher fatality rates, affecting middle-aged adults.Infections caused by strain ST-1466 in the Y subgroup are prevalent, spreading through respiratory secretions or close contact.
The enormous but unknown curative power of old drugsRepurposing existing drugs can lead to breakthrough treatments for rare diseases.Advocacy, research, and collaboration are essential for driving drug development for rare diseases.
New Drug Shows Promise for Treating Rare Brain Tumors - News CenterAn experimental drug, dordaviprone, shows promise in treating H3 K27M-mutant diffuse midline gliomas.The study revealed a 30% positive response rate to the drug in patients with this rare brain tumor subtype.
Is Cord-Blood Banking Worth It?Umbilical cord blood banking can be costly and may not be necessary for all families, especially those without a specific disease history.