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

Delveinsight

AUGUST 17, 2021

Food and Drug Administration, which could have entered the ultra-rare genetic disease market. The rarity of disease – 1.36 Seagen will pay USD 200 million upfront to RemeGen and also secure an exclusive worldwide license agreement of upto USD 2.4 person/million around the globe.

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

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.

Clinical Trials 40

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.

Drugs 40

Drugs Containment Trials Marketing 40

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

Genetics 111

Genetics Vaccination Vaccine DNA 111

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] /.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

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