The Sheridan Press2d
Nonprofit organizes show to support child with Pompe disease
Compete for a Cause will host a 16-act talent competition at 6 p.m. on April 5 at Sheridan College for Pompe Disease patient ...
Amicus Therapeutics Is Very Attractive At Current Prices (Rating Upgrade)
Amicus Therapeutics benefits from strong revenue and limited competition. Learn why FOLD stock is a BUY, with a favorable ...
Scary diagnosis: Young patients in Maine helped to brave their worst medical fears
PORTLAND, Maine (WGME) -- MaineHealth Barbara Bush Children's Hospital is crucial to so many families, some of which are ...
The American Journal of Managed Care6d
For Neuromuscular Disease Community, an Era of Opportunities and Threats
Robert Califf, MD, former commissioner of the FDA, delivered a keynote address at the 2025 Muscular Dystrophy Association ...
Express Healthcare6d
Rare disease treatment: Look at emerging therapies and medications
Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...
Science Daily5d
Groundbreaking AI tool generates 3D map of the brain
As a proof of principle, they generated a 3D interactive atlas of the brain in both normal mice and mouse models of Alzheimer's and Pompe disease, a rare genetic disorder. Sun's lab has made its ...
The Hindu28d
NIMS Medical Genetics Department: A beacon of hope for rare disease patients
Wilson Disease, Phenylketonuria, and Pompe Disease, among others. The NIMS Medical Genetics department was founded in 2008 as the second such department in a government institution in India.
pharmaphorum7d
Astellas licenses companion drug for Pompe gene therapy
currently in the phase 1/2 FORTIS study in late-onset Pompe disease (LOPD) in adults. Xork is designed as a pre-treatment to overcome one of the key limitations to adeno-associated virus (AAV ...
Dyne Therapeutics' $4.3 Billion Peak Sales Potential? Analyst Highlights Key Strengths And Risks
BMO Capital Markets initiated coverage on Dyne Therapeutics, Inc. (NASDAQ:DYN), focused on developing therapies for ...
Amicus Therapeutics, Inc. (FOLD): Among the Stocks Under $10 With High Upside Potential
We recently compiled a list of the 12 Stocks Under $10 With High Upside Potential. In this article, we are going to take a ...
Dyne Therapeutics, Inc.: Dyne Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) to Newly Appointed Chief Financial Officer
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically ...