Every few weeks, Derek Carter gets an e-mail that brings a tear to his eye. Parents around the world have contacted the Carter family seeking more information on Batten’s Disease, a rare, fatal degenerative neurological disorder with no cure. “It makes you cry because I already know what’s going to...
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In many ways, Andrew Kriebel of DuBoistown is like any other 8-year-old boy. He loves jumping on his trampoline, swinging and spending time with his family. But unlike most other children, Kriebel suffers from a rare neurological disorder known as Angelman Syndrome, which makes him prone to seizures, keeps him...
Ashley Howard takes her 5-year-old daughter Gabby to see Oak Island pediatrician Jugta Kahai at least every other week. It can be as often as every week depending on Gabby, who has the extremely rare, genetic disorder primary ciliary dyskinesia that gives her constant ear, sinus and respiratory infections. More...
LYON, France – POXEL SA (Euronext: POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced that Elizabeth Woo has joined the Company as Senior Vice President, Investor Relations, Public Relations and Corporate Communications,...
Washington, DC – PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina. With this funding commitment, we aim to support RDR’s creation of an avenue that increases patient access...
Amsterdam, Netherlands – Worldwide, an estimated, 40 million people live with HIV. Two-thirds of this group on the African continent. In 2022, more than 600,000 people died from HIV-related causes and more than 1.3 million were infected. There is no vaccine against the world’s second most deadly infection, after TB....
CAMBRIDGE, Mass. — Prize4life and the Alzheimer Research Forum announce the release of the ALS Forum (www.ResearchALS.org), a web-based resource for researchers interested in amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease or motor neuron disease. The site is modeled on the Alzheimer Research Forum’s popular website, www.alzforum.org,...
LEIDEN, Netherlands & CAMBRIDGE, Mass. — ProQR Therapeutics NV, a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing oligonucleotides...
Washington, DC – The FDA will host Rare Disease Day, a virtual public meeting, on March 1, 2024 in global observance of Rare Disease Week. This year’s Rare Disease Day is dedicated to patients and health care professionals. Panels will discuss: The legal framework for approving studies and medical products...
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings highlight the need for...