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BioPharma Media

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  • Topic
    • COVID-19
    • New Drugs
    • Oncology
    • Metabolism
    • Neuroscience
    • Autoimmune Diseases
    • Infections
    • Rare/Orphan Diseases
    • Drug Safety
  • Section
    • Pharma & Biotech
    • Regulatory
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Rare/Orphan Diseases

brain
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Relyvrio/Albrioza: New Drug for Amyotrophic Lateral Sclerosis Treatment

September 29, 2022September 30, 2022

Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.

gene therapy
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy

September 19, 2022September 19, 2022

A single dose of the elivaldogene autotemcel will cure a deadly disease.

blood gene therapy
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Roctavian: Gene Therapy for Hemophilia A

September 13, 2022September 13, 2022

A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

September 13, 2022September 13, 2022

One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.

multiple-myeloma
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Tecvayli: New Drug for Multiple Myeloma

August 24, 2022September 8, 2022

Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.

lungs
Posted inClinical Trials, Rare/Orphan Diseases

Wynayre/Wynfidra: New Drug for COPD Maintenance Therapy

August 9, 2022September 8, 2022

Ensifentrine, an inhaled PDE3/PDE4 inhibitor, in the treatment of chronic obstructive pulmonary disease.

brain
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases

Qalsody: New Drug for Amyotrophic Lateral Sclerosis with SOD1 Mutation

August 2, 2022September 8, 2022

Tofersen and the mixed results of treatment for the deadly neurodegenerative disease.

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