XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

Intouch Solutions

APRIL 11, 2024

Pre-Clinical : Deeper comprehension of the impacts and outcomes associated with a particular drug. Clinical : Optimized efficiency, inventory management, and service quality of clinical trials. This can make a difference in all cases, but particularly with rare diseases.

Compliance 52

Compliance Marketing Drugs Pharma Companies 52

Rethinking Clinical Trials

JANUARY 23, 2024

Similar to most types of trials, there are pros and cons to both pragmatic and explanatory designs in imaging trials. People with angina-like symptoms are often not patients with a disease. Most do not have obstructive coronary artery disease (CAD), but a few are very high risk. There has only been a hand full of trials.

Trials 154

Trials Cardiology Clinical Trials Clinical Trials 154

XTalks

AUGUST 19, 2022

But bluebird bio has said such setbacks are expected for a company at the frontier of developing gene therapies for rare diseases. The most severe form of the disease is sometimes called transfusion-dependent beta-thalassemia or beta-thalassemia major. Zynteglo’s hefty price tag of $2.8

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). CALD is a rare, progressive neurodegenerative disease that mainly affects young boys. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

XTalks

DECEMBER 21, 2022

This included greater adoption and acceptance of decentralized clinical trial models, emphasis on patient-centric approaches in trials, growing digitization of healthcare and dealing with drug manufacturing challenges. There is also generally a low cost of return for gene therapies, particularly those for rare diseases.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

pharmaphorum

AUGUST 10, 2020

Evrysdi is the third treatment approved for SMA , an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec). Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

FDA Approval 59

FDA Approval Drugs Gene Therapy Protein 59

XTalks

MARCH 22, 2023

The years that followed have seen a rise in large pharma companies acquiring much smaller biopharmas with a narrow focus on rare disease therapeutic development. The proton-pump inhibitor is designed to reduce stomach acid levels, thereby treating digestive conditions like gastroesophageal reflux disease (GERD).

Life Science 97

Life Science Drugs FDA Approval Vaccination 97

The Pharma Data

MAY 4, 2021

In addition, we have achieved important clinical, regulatory and commercial milestones across our pipeline and portfolio while also continuing to increase our capacity to supply urgently-needed doses of BNT162b2 to the world. Rare Disease. Clinical and Research Developments. Internal Medicine. Total Revenue.

Vaccination 40

Vaccination Vaccine Production Sales 40