rare disease Research Success in Rare Disease / RHEACELL receives positive signal for accelerated stem cell development program in rare Butterfly Disease' By www.prleap.com Published On :: Wed, 28 Feb 2024 06:00:00 PST On February 29, 2024, Rare Disease Day will take place worldwide to raise awareness for rare diseases. Only 5% of the approximately 6,000 to 10,000 known rare diseases are currently treatable. The research and development of targeted therapeutic approaches is time-consuming, so that many companies shy away from the financial outlay in view of the low number of patients. Full Article
rare disease On Rare Disease Day 2nd Chapter Productions Announces Unheard: The Ears of Meniere's Documentary By www.24-7pressrelease.com Published On :: Mon, 27 Feb 2023 08:00:00 GMT Filming Begins With Tech-Evangelist Guy Kawasaki & Dr. Kevin Peng of The House Institute Foundation With Support from Former Tampa Bay Buccaneer Mike Liedtke Full Article
rare disease Andarix Pharmaceuticals to Present at Clinical Trials in Rare Diseases Conference 2022 By www.24-7pressrelease.com Published On :: Thu, 08 Dec 2022 08:00:00 GMT Personalized and targeted medicine for rare lung and other cancers Full Article
rare disease Protheragen Introduces Disease Model Development Platform to Empower Rare Disease Therapy Research By www.24-7pressrelease.com Published On :: Sun, 13 Oct 2024 08:00:00 GMT Protheragen announced its disease model development platform to develop 2D cell culture, 3D organoid, and animal models for researchers. Full Article
rare disease Proteomic signatures improve risk prediction for common and rare diseases - Nature.com By news.google.com Published On :: Mon, 22 Jul 2024 07:00:00 GMT Proteomic signatures improve risk prediction for common and rare diseases Nature.com Full Article
rare disease Delaware Rare Disease Advisory Council Hosts First Meeting By news.delaware.gov Published On :: Thu, 27 Jun 2024 16:12:13 +0000 DOVER, Del. — The Delaware Rare Disease Advisory Council held its inaugural meeting on Tuesday, taking its first steps to address innovation and research, patient care, and affordable and accessible treatment therapies. Delaware is one of 27 states with a Rare Disease Advisory Council. Established by Senate Bill 55, sponsored by Sen. Nicole Poore […] Full Article Lt. Governor Bethany Hall-Long News Office of the Lieutenant Governor
rare disease Looking for Opportunities to Accelerate Clinical Research in Rare Diseases By lifescivc.com Published On :: Wed, 17 Jul 2024 11:00:40 +0000 By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets. Identifying The post Looking for Opportunities to Accelerate Clinical Research in Rare Diseases appeared first on LifeSciVC. Full Article Business Development From The Trenches Portfolio news Rare Diseases Science & Medicine CFTR Cystic Fibrosis NBD1 Sionna Therapeutics
rare disease Rs 50 Lakh Lifeline for Rare Disease Patients By www.medindia.net Published On :: Union Minister of State for Health and Family Welfare, Anupriya Singh Patel, announced that up to Rs. 50 lakhs per patient has been allocated for the Full Article
rare disease Shire Receives Breakthrough Therapy Designation for Rare Diseases Drugs By www.medindia.net Published On :: US Food and Drug Administration has granted breakthrough therapy designation for two experimental drugs for rare diseases conducted by the biotechnology company Shire. Full Article
rare disease Stop abuse of patent monopolies in rare diseases, say patient representatives and treatment activists By www.thehindu.com Published On :: Tue, 05 Nov 2024 19:17:18 +0530 They seek urgent intervention by government, courts, and lawmakers into the issue Full Article Health
rare disease Rare diseases patient representatives seek govt intervention to access treatment By www.thehindubusinessline.com Published On :: Tue, 05 Nov 2024 19:30:48 +0530 Monopoly could hinder Union Health Ministry’s efforts “to benefit from domestic production and competitive pricing through pooled procurement strategies” Full Article National
rare disease The challenges in testing and treatment of rare diseases By www.thehindu.com Published On :: Fri, 22 Sep 2023 07:00:00 +0530 Treatment for six rare disease patients in Karnataka, under the Centre’s grant of ₹50 lakh each, was stopped recently because the funding limit had been reached. Afshan Yasmeen The Hindu speaks to patients and families and takes an overview of the treatment scenario Full Article Health
rare disease What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis By pediatrics.aappublications.org Published On :: 2012-02-27T00:08:27-08:00 Rare diseases in childhood can be debilitating and require lifelong care. Since 1983, the Orphan Drug Act incentives have stimulated the development and significantly improved the availability of treatment products for patients with rare diseases.We report an increasing pediatric orphan product designations and approvals from 2000 to 2009. The trend indicates that the Orphan Drug Act has continued to address this important unmet need. (Read the full article) Full Article
rare disease 5-Year-Old In New York Dies From Rare Disease Linked To COVID-19 By www.ndtv.com Published On :: Fri, 08 May 2020 23:08:56 +0530 A five-year-old boy in New York state has died from a rare inflammatory disease believed to be caused by the new coronavirus, Governor Andrew Cuomo said Friday. Full Article World
rare disease New York child dies from rare disease linked to Covid-19 By feedproxy.google.com Published On :: Sat, 09 May 2020 06:00:00 +0500 NEW YORK: A five-year-old boy in New York state has died from a rare inflammatory disease believed to be caused by the new coronavirus, Governor Andrew Cuomo said on Friday.“There have been 73 reported cases in NY of children getting severely ill with symptoms similar to Kawasaki disease... Full Article
rare disease A quick reference guide for rare disease: supporting rare disease management in general practice By bjgp.org Published On :: 2020-04-30T16:04:41-07:00 Full Article
rare disease Boy, 5, in New York dies of rare disease linked to coronavirus By www.telegraph.co.uk Published On :: Sat, 09 May 2020 01:02:56 GMT Full Article topics:places/usa structure:news/world-news structure:news topics:places/new-york-city topics:in-the-news/coronavirus storytype:standard
rare disease PTC Therapeutics to Acquire Censa Pharma for Mid-Stage Rare Disease Drug By xconomy.com Published On :: Thu, 07 May 2020 07:09:51 +0000 PTC Therapeutics has reached an agreement to acquire Censa Pharmaceuticals and its lead asset, an experimental metabolic disorder therapy that is now being prepared for a pivotal study. The deal, announced after the market close Wednesday, will bring South Plainfield, NJ-based PTC (NASDAQ: PTCT) another compound for its pipeline of rare disease drugs. Wellesley, MA-based […] Full Article Boston blog main Boston top stories National blog main New York New York blog main New York top stories Arkin Bio Ventures BioMarin Pharmaceutical Biotech Censa Therapeutics clinical trials Drug Development FDA Life Sciences PTC Therapeutics rare disease
rare disease Boy With a Rare Disease That Covered Him in Blisters, and Burns Fights For Life By feedproxy.google.com Published On :: Five-year-old Ollie Williams went down suddenly by a rare disease called Stevens-Johnson syndrome this May. This disease caused a sudden outburst of blisters Full Article
rare disease New Gene for Rare Disease of Excess Bone Growth Identified By www.medindia.net Published On :: New gene that causes melorheostosis, a rare group of conditions involving painful and disfiguring overgrowth of bone tissue, is identified by the team Full Article
rare disease Physician-Parents Whose Children Have Rare Diseases By jamanetwork.com Published On :: Tue, 05 May 2020 00:00:00 GMT In this essay, a critical care pediatric hospitalist finds herself on the other side of the office table advocating for the specific medical care needed to address her son’s rare skeletal dysplasia and her search for a pediatric specialist with whom to travel on this quest. Full Article
