Shropshire Star9h
Boy, eight, with rare disease completes Ironkids Wales with walker
The mother of an eight-year-old boy with a rare disease has spoken of her pride at seeing him complete Ironkids Wales with a ...
TMCnet3d
Larimar Therapeutics Announces Three Poster Presentations at the Upcoming International Congress for Ataxia Research
Larimar will present three posters during the conference, one of which will also be presented as an oral presentation. The poster presentations are as follows: Oral presentation by Dr. Russell Clayton ...
Yahoo Finance3d
Liquidia Corporation (LQDA)
16, 2024 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA) (Liquidia), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary diseases ...
Woman with rare disease claims she’s now blind and bruised after doctors made her get 3 vaccines
A 23-year-old Florida woman has been temporarily blinded and bruised after allegedly being required to receive multiple ...
STAT3d
House panel passes pediatric rare disease bill, setting it up for government funding bill
A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in ...
STAT10d
A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
After his son Michael was diagnosed with a devastating, ultra-rare disease in 2019, Terry Pirovolakis dedicated himself to eradicating it. Pirovolakis, a Canadian IT director, recruited ...
CNN9d
Pro-Russian posters appear on billboards across Italy
Hundreds of posters have appeared on billboards across Italy this summer, bearing the slogan: “Russia is not our enemy” and depicting a handshake in the colors of the Italian and Russian flags.
Health officials in L.A. Co. investigating rare disease spread from raccoons to humans
The Los Angeles County Department of Public Health is looking into two human cases of a rare parasitic infection that comes ...
FierceBiotech9d
FDA pushed to be more open, collaborative to accelerate rare disease R&D
The FDA should be more open and collaborative to unleash a surge in approvals of rare disease drugs, according to a report by the National Academies of Sciences, Engineering, and Medicine.
Medical Xpress10d
How parents and caregivers can evaluate the research on MERT and other potential autism treatments
"It's not that impactful from a scientific perspective." Many MERT clinics feature a 2014 electronic poster presentation that examines data from the charts of 141 children who received ...
Yahoo Finance26d
North America Rare Disease Therapeutics Market Size, Trends, Competition, Forecasts & Opportunities, 2019-2023 & 2024-2029
Dublin, Aug. 27, 2024 (GLOBE NEWSWIRE) -- The "North America Rare Disease Therapeutics Market, By Country, Competition, Forecast & Opportunities, 2019-2029F" report has been added to ...
National Academies of Sciences%2c Engineering%2c and Medicine10d
To Increase Pace and Volume of Drug Approvals for Rare Diseases, Report Recommends FDA Enhance Information Sharing, Improve Collaboration
WASHINGTON — The Food and Drug Administration should enhance information sharing, advance the regulatory science needed to evaluate rare disease drugs, improve engagement with people with rare ...