Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
Rare/Orphan Diseases
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy
A single dose of the elivaldogene autotemcel will cure a deadly disease.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Roctavian: Gene Therapy for Hemophilia A
A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Tecvayli: New Drug for Multiple Myeloma
Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.
Posted inClinical Trials, Rare/Orphan Diseases
Wynayre/Wynfidra: New Drug for COPD Maintenance Therapy
Ensifentrine, an inhaled PDE3/PDE4 inhibitor, in the treatment of chronic obstructive pulmonary disease.
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases
Qalsody: New Drug for Amyotrophic Lateral Sclerosis with SOD1 Mutation
Tofersen and the mixed results of treatment for the deadly neurodegenerative disease.
