The Rarest of All Diseases Are Becoming Treatable

This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.
BioSpace

Rare Disease Leaders Call for Regulatory Consistency After Chaotic Year

As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a ...
Becker's Hospital Review

Lurie Children’s receives $11M gift to launch genetics and rare diseases division

Ann & Robert H. Lurie Children’s Hospital in Chicago has received an $11 million gift to launch a division focused on genetics and rare diseases. The gift from Don and Anne Edwards will create the ...
The Mirror US on MSN

Dad diagnosed with rare disease after dismissing aches and pains as 'aging'

Robin Schultz, 35, discovered he had Fabry disease after putting his son through genetic testing when the toddler wasn't ...
Fierce Pharma

Italian nonprofit lands FDA approval for first gene therapy to treat rare genetic disease

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

Lurie Children's Hospital receives $11 million gift for genetics and rare disease research

Ann & Robert H. Lurie Children's Hospital on Wednesday announced what they called a "transformative gift," as investment banker Don Edwards and his wife, Anne, have donated $11 million to the hospital ...
Clinical Trials Arena on MSN

Utilising real-world data to overcome challenges in rare disease research

Synthetic control arms may help tackle the unique complexities, but key challenges such as data access and standardisation ...
Good News Network

Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

Covid brought rare-disease drug research to a halt. It still hasn't recovered.

The Covid-19 pandemic brought rare disease research to a standstill. Five years later, patients and advocates say the ...
HME News

Soleo Health to broaden rare disease portfolio with new exec

Soleo Health has appointed Jody Thompson as senior vice president of business development, rare disease, to broaden the company’s portfolio of exclusive and limited distribution therapies for rare, ...

Country music star dead after battle with LMD. What we know about the rare disease

Beloved country music singer Raul Malo has died after a long battle against stage 4 cancer and leptomenigeal disease. His group, The Mavericks, shared the news Tuesday on Instagram. “It’s with the ...