Pharmaceutical Technology

DECEMBER 5, 2022

Cannabinoid receptors are a popular therapeutic target for cannabinoid-based drugs in the treatment of pain, neurological disorders and inflammation, according to GlobalData’s Pharma Intelligence Centre Drugs database. This is closely followed by CB2 receptors in second place.

Regulation 245

Regulation Drugs Development Marketing 245

Pharmaceutical Technology

FEBRUARY 10, 2023

This is not the first treatment to come with a high price tag. However, more highly innovative/high-cost treatments, within infectious disease and neurology space, are likely to enter the market for rare disorders, increasing the number of non-oncology RSAs.

Gene Therapy 263

Gene Therapy Marketing Gene Drugs 263

XTalks

DECEMBER 13, 2023

Sickle cell disease is a group of inherited red blood cell disorders that affect approximately 100,000 people in the US, most of whom are Black. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Gene Silencing Gene Clinical Trials Clinical Trials 98

XTalks

OCTOBER 12, 2022

For ASL MR perfusion, water molecules in arterial blood from a feeder artery are magnetically tagged and tracked until they reach the region of interest. Then, the signal difference in the region of interest between the tagged images and control images can be computed to obtain cerebral blood flow (CBF) values ( Figure 3 ).

Clinical Trials 93

Clinical Trials Clinical Trials Trials Drugs 93

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). It is specifically indicated to slow the progression of neurologic dysfunction in boys four to 17 years of age with early, active CALD. Related: Bluebird’s $2.8M

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FDA Approval Gene Therapy Drugs Gene 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. With the approval, Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy also became the first CRISPR/Cas9-based therapy approved in the US.

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Research Drugs Gene Therapy Clinical Trials 59