PQA

FEBRUARY 29, 2024

Today is Rare Disease Day. It’s an opportunity to raise awareness of the impact of rare diseases. There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. In the United States, rare disease affects about one in 10 people.

Specialty Pharmacies 45

Specialty Pharmacies Communication Patient Care 45

pharmaphorum

FEBRUARY 26, 2021

Rare diseases affect a fraction of the population – 450 million + people globally. However, its effects can be equally severe and life-threatening as other prevalent diseases. For Rare Disease Day, Tarun Kumar Bansal discusses why we need to expedite innovation in the rare disease domain.

Compounding 58

Compounding Hospitals FDA 58

PharmaShots

JANUARY 31, 2023

Shots: Immutable capital demands and high-spending drug development plans are the most common drivers in encouraging fully-fledged private healthcare companies to go public. The lead program of the company, THIO, is an investigational dual mechanism of action drug incorporating telomere targeting and immunogenicity in patients with NSCLC.

Vaccines 40

Vaccines Immunization FDA 40

Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. Cablivi stops platelets from clumping together, which prevents small clots, and can be used along with other drugs that suppress the immune system.

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Documentation Immunization Pharmaceutical Companies Pharmaceutical Companies 52