As reported on Healio, the U.S. Food and Drug Administration has approved finerenone (Kerendia; Bayer) for adults with chronic kidney disease (CKD) associated with type
To start, a few words about the immune system: The immune system is comprised of cells, molecules, and biochemicals, and works to maintain human health.
Accessible travel planning means building a trip around a traveler’s actual mobility, sensory, medical, communication, or support needs—not simply choosing destinations labeled “accessible.” A successful
For new caregivers supporting a parent, partner, or relative at home, the days can turn into an unbroken string of decisions, vigilance, and worry. The
When I was 39 years old, my life changed in an instant. I was a father of two young children, my daughter was six and
As reported on News-Medical, an experimental treatment for idiopathic pulmonary fibrosis (IPF), rentosertib, was associated with shifts toward younger biological age profiles across multiple protein-based
As reported on PharmaBiz, the U.S. Food and Drug Administration has accepted Takeda’s New Drug Application for the investigational oral therapy zasocitinib (TAK-279) and assigned
My name is Terri, and I live in the small town of Searcy, Arkansas, where I’ve proudly connected with countless people and their families over
As reported on Fierce Biotech, the U.S. Food and Drug Administration (FDA) has launched a new pilot program designed to streamline the investigational new drug
Editor’s Note: This story was originally written and submitted to us by Anthony Clark. “Okay, Michael, hold the ball up to your ear, then throw
The condition Dupuytren disease, also called Dupuytren contracture, is a chronic disorder of connective tissue in the palm. Tissue gradually becomes thickened and tight, forming
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has authorized Acepodia to proceed with clinical testing of ACE723, an investigational antibody-drug conjugate
As reported on Fierce BioTech, a phase 3 study conducted by MediLink has delivered positive results for Roche-partnered antibody-drug conjugate (ADC) tambotatug pelitecan (tam-peli), also
What’s the Good News? Researchers have successfully completed initial testing of an experimental Parkinson’s medication called TT-P34. The early results are very encouraging and suggest
The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the
This is my son Nikolai. Nikolai was recently diagnosed with Mucopolysaccharidosis type 2, or “Hunter Syndrome.” A rare genetic form of childhood dementia, it’s caused
As reported on PharmaBiz, a combination of Imfinzi (durvalumab) and Imdelltra (tarlatamab) has delivered significant improvements in survival outcomes for patients with extensive-stage small cell
At the age of 18, I was diagnosed with a rare swallowing disorder called Achalasia. At the time of this video, I had undergone several
Cure GM1 FoundationDecades of rare disease expertise, all pointed at a single goal: advancing enzyme replacement therapy for GM1 to clinical trials. The team is in place. The science is ready. What they need now is the funding to finish.
Meet the team and the mission: curegm1.org/ert-project Join the fight: curegm1.org/donate
#CureGM1 #GM1Gangliosidosis
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