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Results For "rare-diseases"

109 News Found

US FDA grants Soligenix Orphan Drug Designation for the treatment of T-cell lymphoma
Drug Approval | September 12, 2021

US FDA grants Soligenix Orphan Drug Designation for the treatment of T-cell lymphoma

Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma


US FDA grants Lantern Pharma additional Orphan Drug tag for LP-184
Biotech | August 31, 2021

US FDA grants Lantern Pharma additional Orphan Drug tag for LP-184

The drug is also intended for the treatment of glioblastoma multiforme


European Commission grants market authorisation for BioMarin’s Voxzogo
Biotech | August 28, 2021

European Commission grants market authorisation for BioMarin’s Voxzogo

It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with Voxzogo


Apollo Children’s hospital saves new born with rare brain vascular malformation
Healthcare | August 18, 2021

Apollo Children’s hospital saves new born with rare brain vascular malformation

Seen in 1 in 3 million births, the malformation was detected at the antenatal stage during the mother's pregnancy


USFDA grants distinct indication for Chronic Sleep Disorder
Drug Approval | August 13, 2021

USFDA grants distinct indication for Chronic Sleep Disorder

Idiopathic hypersomnia is a life-long condition, and the approval of Xywav will be instrumental in providing treatment for symptoms such as excessive sleepiness and difficulty waking, and in effectively managing this debilitating disorder


Fulgent Genetics and Helio Health partners to commercialize early cancer detection tests
Digitisation | August 10, 2021

Fulgent Genetics and Helio Health partners to commercialize early cancer detection tests

Partnership enables broad reach of cfDNA methylation blood tests for early cancer detection


FDA approves Nexviazyme for late-onset Pompe disease
Drug Approval | August 09, 2021

FDA approves Nexviazyme for late-onset Pompe disease

Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells


Sanofi to acquire Translate Bio
Biotech | August 07, 2021

Sanofi to acquire Translate Bio

Advances deployment of mRNA technology across vaccines and therapeutics development


Lonza announces expansion plans for mammalian manufacturing facilities
News | May 07, 2021

Lonza announces expansion plans for mammalian manufacturing facilities

The facility is expected to be completed in 2024