BioSpace

Despite Wide Support for Rare Disease, Voucher Program Caught Up in Senate’s ICE Fight

The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding ...

This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

Why my daughter’s rare genetic disorder is CT’s moral imperative

The crisis facing rare disease and disability families in Connecticut is caused by systemic underinvestment, administrative barriers, and policy choices.
Morning Overview on MSN

Ancient DNA uncovers 12,000-year-old case of rare genetic disorder

A teenager who lived in southern Europe around 12,000 years ago has become the earliest person in history to receive a ...
WALB

Six-year-old South Georgia girl faces rare bone disorder with unshakable strength

To be only six years old and have seven breaks and two surgeries, that’s a lot,” Jessica said. “Some days are really hard.” ...
BioSpace

Rare Disease Biotechs Stand To Lose $4B if Priority Voucher Program Not Reinstated: Report

Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...
Fierce Biotech

Moderna offloads late-stage rare disease drug to Recordati for $50M upfront

Moderna has outlicensed a rare disease drug candidate to Recordati, accepting $50 million upfront for an asset that could deliver registrational data this year.

Jaguar Health Highlights Sharp Strategic Focus on Rare Intestinal Failure Diseases Fueled by Non-Dilutive Funds from Closing of License Deal for Mytesi

Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
The Manila Times

CIRM approves new $100 million plan to accelerate genetic therapies for patients with rare diseases

The California Institute for Regenerative Medicine (CIRM) has approved a new funding program to accelerate therapies for rare diseases, which sets aside $100 million over two years to create a ...

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for an Additional Dosing Regimen of Every Four Weeks for Elfabrio® (pegunigalsidase alfa ...

Committee for Medicinal Products for Human Use (CHMP) issues a positive opinion following re-examination, which will be reviewed by the European Commission (EC), with a decision anticipated by March 2 ...
Morningstar

National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide

NORD's growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration With these new members, the network now ...