Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...
Human case of rare insect-borne disease reported in Boulder County
Boulder County is reporting its first human case of tularemia in 2024, following a case of tularemia in a Jefferson County ...
pharmaphorum1d
Patient Recruitment for Rare Disease Trials Summit
Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...
STAT6d
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 ...
Developing tailor-made treatments for ultra-rare disease patients: Research provides a roadmap
In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...
Science News1d
A common drug may help treat a rare genetic disease
Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...
Tahlequah Daily Press2d
Cherokee Nation to host Rare Disease Summit in Tahlequah Nov. 21
The Cherokee Nation is hosting a Rare Disease Summit Nov. 21 to address the unique challenges facing tribal citizens with rare medical conditions. The event, organized by Cherokee Nation Health ...
The American Journal of Managed Care2d
Contributor: The Critical Role of Patient Engagement in Patient-Centered Outcomes Research and Health Technology Assessment of Rare Disease Treatments
Patient engagement is a necessity when performing health technology assessment to help better assess and identify the outcomes of a rare disease.
FiercePharma5d
Arrowhead targets rare disease patients, pushing message that hope is on the horizon
Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...
Pulmonaryhypertensionnews.com3d
Rare disease advocacy is a year-round endeavor for me
While PH Awareness Month is a great opportunity to connect and educate, rare disease advocacy is columnist Jen Cueva's passion all year.