
FDA Approves Fayuvi, the First Gene Therapy for Children with Sanfilippo Syndrome Type A
One-time treatment provides a new option for pediatric patients with a rare, progressive genetic disorder As announced in a recent press release, the U.S. Food

One-time treatment provides a new option for pediatric patients with a rare, progressive genetic disorder As announced in a recent press release, the U.S. Food

I want to share this to give some ideas I have found that help me move through difficult days with more steadiness and self compassion,

As reported on Radiology Business, the American Society for Radiation Oncology (ASTRO) has released its inaugural clinical practice guideline dedicated to the use of radiation

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
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From our friends @EpilepsyFoundationofVirginia #SupportGroup#TomorrowPlease join us for the monthly Epilepsy Support Group this Wednesday, Sept 23rd at 7:00pm! Participation is virtual. Scan the QR code to join the Zoom, or check the image for Zoom ID and password.
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