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.
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 ...
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 ...

Rezolute's rare disease drug fails to meet main goal in late-stage trial

Rezolute said on Thursday its experimental drug for a rare genetic condition that causes dangerously low blood sugar failed ...

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 ...
STAT

In a first, a nonprofit wins FDA approval for rare disease gene therapy

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Crain's Chicago Business

Philanthropists give $11 million for Lurie Children's rare genetic disease program

Don and Anne Edwards have donated $11 million to Ann & Robert H. Lurie Children's Hospital to establish what will be the ...

Lurie Children's to expand research in fight against rare genetic diseases thanks to $11M donation

Lurie Children's Hospital officially announced an expansion of research with the $11 million from Don and Anne Edwards, who ...

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 ...
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, ...