Infrequent – Page 2

Neurogene Halts Development of Batten Gene Therapy NGN-101 Following FDA Denial

5 months ago talkbio0Tagged Denial (Psychology), disease research, gene therapy, Infrequent, Juvenile Neuronal Ceroid Lipofuscinosis, Neurogene, NGN-101, United States Food and Drug Administration

Neurogene, Batten disease, gene therapy, NGN-101, FDA denial, rare disease research

Intellia’s CRISPR Gene Editing Therapy Shows Promising Results in Reducing HAE Attacks

6 months ago talkbio0Tagged Angioedemas, Hereditary, Clinical Trials, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, gene therapy, Infrequent, Intellia

Intellia Therapeutics, CRISPR Gene Editing, Hereditary Angioedema (HAE), NTLA-2002, Clinical Trials, Gene Therapy, Rare Diseases

Sanofi Invests €300M in Orano Med for Next-Generation Radioligand Therapies

6 months ago talkbio0Tagged 212Pb, AlphaMedix, Infrequent, Malignant Neoplasms, Multiple Epiphyseal Dysplasia, radioligand therapies, sanofi, targeted alpha, targeted alpha therapies

Sanofi, Orano Med, Radioligand Therapies, Rare Cancers, AlphaMedix, Lead-212 (212Pb), Targeted Alpha Therapies

UK Regulator Approves Pharming’s Joenja for Rare Immune Disorder APDS, Leveraging FDA Review

7 months ago talkbio0Tagged APDS, Approved, Immune System Diseases, Infrequent, Joenja, leniolisib, Medicines And Healthcare Products Regulatory Agency, Molecular Farming, pamidronate, Review [Publication Type], United States Food and Drug Administration

Joenja, leniolisib, APDS, MHRA approval, FDA review, rare immune disorder, Pharming

Biohaven Revives Troriluzole Hopes with Positive Update for Spinocerebellar Ataxia

7 months ago talkbio0Tagged Ataxia, Spinocerebellar, Clinical Trials, Infrequent, Neurodegenerative Disorders, New Drug Application, SCA, Troriluzole, United States Food and Drug Administration

Troriluzole, Spinocerebellar Ataxia (SCA), Biohaven Pharmaceuticals, Neurodegenerative Disorders, FDA New Drug Application (NDA), Clinical Trials, Rare Diseases

Zevra Therapeutics Secures FDA Approval for Miplyffa, a Breakthrough Treatment for Niemann-Pick Disease Type C

7 months ago talkbio0Tagged Approved, Infrequent, Lysosomal Storage Diseases, Miplyffa, Niemann-Pick Disease, Type C, United States Food and Drug Administration

Zevra Therapeutics, Miplyffa, FDA Approval, Niemann-Pick Disease Type C, Rare Lysosomal Storage Disorder, Arimoclomol

AstraZeneca Secures FDA Approval for Fasenra in Rare Autoimmune Disorder EGPA

7 months ago talkbio0Tagged Approved, Asthma, AstraZeneca, Autoimmune Diseases, biology (field), Eosinophilic granulomatosis with polyangiitis, Infrequent, United States Food and Drug Administration

AstraZeneca, Fasenra, FDA approval, EGPA, rare autoimmune disorder, asthma biologic

AstraZeneca’s Fasenra Gains FDA Approval for Eosinophilic Granulomatosis with Polyangiitis, Expanding Treatment Options for Rare Autoimmune Disease

7 months ago talkbio0Tagged Approved, asthma medication, AstraZeneca, Autoimmune Diseases, biology (field), eosinophilic, Eosinophilic granulomatosis with polyangiitis, Granulomatosis, GSK 's, Infrequent, Nucala, Systemic Vasculitis, United States Food and Drug Administration

AstraZeneca, Fasenra, FDA approval, eosinophilic granulomatosis with polyangiitis (EGPA), rare autoimmune disease, biologic treatment, asthma medication, GSK’s Nucala