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.

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Protein RNA Production Genetics 52

The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency – The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S.

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Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

NOVEMBER 2, 2020

Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia. BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. In the U.S., About BioMarin.

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The Pharma Data

OCTOBER 28, 2020

GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. About Ultragenyx.

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Protein Production Development Drugs 40

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.

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Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Source link: [link] /.

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Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

DECEMBER 13, 2023

Xoma’s unique approach involves acquiring future economic interests tied to pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotech companies. It is now progressing towards the submission of a Biologics License Application (BLA) by the end of 2023. million, compared to $1.1

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Genetics Vaccine Vaccination DNA 111