STAT5d
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 ...
ABP Live on MSN1d
This Rare Disease Catches Infants Within First 6 Months, Could Go Unnoticed Until It’s Too Late
By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.
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.” ...
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 ...
FiercePharma4d
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 ...
BioCryst’s Promising Prospects in Rare Disease Market Driven by BCX17725 and Orladeyo Success
In a report released today, Andrew Fein from H.C. Wainwright reiterated a Buy rating on BioCryst (BCRX – Research Report), with a price ...
Tahlequah Daily Press2d
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 ...
JD Supra2d
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub
Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...
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.
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 ...
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 ...