FierceBiotech18h
ElevateBio gene therapy lowers levels of toxic protein in mice with Huntington's disease
ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...
GlobalData on MSN16h
Inborn errors of metabolism elicit unique challenges for therapy development
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
Healio17h
European Medicines Agency recommends ‘landmark’ gene therapy for epidermolysis bullosa
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...
UMass Study: Gene Therapy Hope for Maple Syrup Disease
A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented ...
Santa Rosa Press Democrat11h
Ultragenyx Pharmaceutical revenue up 29% in 2024
The Novato-based maker of biopharmaceutical treatments for rare diseases plans to release this year a new gene therapy for ...
pharmaphorum21h
On incentivisation and investment in rare disease R&D
There is huge unmet need for new rare disease therapies, where patients have few or no available options. According to the ...
Sheffield Teaching Hospitals recruits world’s first patients into revolutionary gene study for rare muscle disorder
A 24-year-old woman from York has become one of the first patients in the world to take part in a pioneering research study ...
Clinical trial provides hope for Mass. child with rare genetic disorder
On the very day Noa was diagnosed, an experimental gene therapy and clinical trial were posted to treat Canavan disease. Noa was only the third patient in the world to be dosed with the gene therapy, ...
GlobalData on MSN23h
Why pharma is still far from a treatment for galactosemia
With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are ...
BioSpace20h
6 Rare Diseases That Have New FDA-Approved Treatments
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...
pharmaphorum23h
From discovery to delivery: Fast-tracking patient access to life-saving rare disease treatments
Other companies use customer reference and patient data to improve operational agility as they launch and scale their first ...
Krystal Biotech Receives Positive CHMP Opinion for VYJUVEK® for the Treatment of Dystrophic Epidermolysis Bullosa
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal ...