XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

JANUARY 27, 2023

Minimising any of those delays that happen throughout the clinical trial, or the clinical development is really important and, more and more, it’s about having experts involved in providing advice in that clinical development and clinical trial programme [that] is really important.”

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

The Pharma Data

NOVEMBER 11, 2020

Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. H1 2021: Initiation of Phase 1/2 clinical trial. Demetrios Braddock, M.D.,

Clinical Trials 40

Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

Sales 52

Sales Marketing Containment Development 52

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

The Pharma Data

OCTOBER 28, 2020

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

Protein 40

Protein Production Development Drugs 40