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Rare/Orphan Diseases

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

by Mark Gubar 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

by Mark Gubar August 24, 2022September 8, 2022

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

brain
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases

Qalsody: New Drug for Amyotrophic Lateral Sclerosis with SOD1 Mutation

by Julia Mardi August 2, 2022April 1, 2024

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

Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases

Alsitek: New Drug for ALS

by Mark Gubar and Julia Mardi June 17, 2022September 8, 2022

Oral masitinib doubles the life in patients with amyotrophic lateral sclerosis.

olorofim
Posted inClinical Trials, Infections, Rare/Orphan Diseases

Olorofim: New Antifungal Drug

by Tanya von Reuss June 9, 2022September 8, 2022

Olorophim by F2G and Shionogi & Co. is focused on invasive aspergillosis treatment.

Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Tepezza: First Drug for Thyroid Eye Disease

by Tanya von Reuss April 27, 2022September 12, 2022

Teprotumumab by Horizon Therapeutics can really help with proptosis and diplopia in Graves’ disease and beyond.

lymphatic vessels
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Vijoice: First Targeted Treatment for PIK3CA-Related Overgrowth Spectrum (PROS)

by Tanya von Reuss April 23, 2022September 8, 2022

Novartis and its alpelisib will help with very rare diseases with malformations and excessive tissue growth.

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