Research

Focus on the pharmaceutical and biotech industry
The Promise of Gene Therapy and the Challenges of Payment Structures

The Promise of Gene Therapy and the Challenges of Payment Structures

Jeff Ajer, Executive Vice President and Chief Commercial Officer of BioMarin, discusses gene therapy. The development of these transformative therapies has triggered discussions about market access challenges, the viability of alternative financing mechanisms, and the results for patient access.

Sanfilippo Syndrome

Sanfilippo Syndrome

Raj Mehra, PhD, Chief Executive Officer of Seelos Therapeutics provides an overview of Sanfilippo syndrome, also known as Mucopolysaccharidosis type III (MPS III), a progressive disorder that primarily affects the brain and spinal cord (central nervous system).

Update of the STARS Study: Sarizotan Treatment of Apneas in Rett Syndrome

Update of the STARS Study: Sarizotan Treatment of Apneas in Rett Syndrome

Stefan Weber, CEO of Newron Pharmaceuticals, provides an update on the “Sarizotan Treatment of Apneas in Rett Syndrome” (STARS) study, a potential pivotal clinical study performed in study centers in the US, Europe, Asia, and Australia to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms.

Key Trends from 15th Annual WORLDSymposium for Lysosomal Storage Disorders

Key Trends from 15th Annual WORLDSymposium for Lysosomal Storage Disorders

Hartmann Wellhoefer, MD, Vice President, Medical Affairs, Rare Disease and Internal Medicine, Global R&D at Takeda sat down with CheckRare to discuss the 15th Annual WORLDSymposium 2019 to share his insights about this year’s conference including key highlights and trends and Takeda’s presence.

Benamin Forred Discusses Rare Disease Day

Benamin Forred Discusses Rare Disease Day

Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses Rare Disease Day and his organization which is one of the largest health systems in the United States, including research into rare diseases.

Gene Testing for Inherited Eye Diseases

Gene Testing for Inherited Eye Diseases

Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses gene testing for inherited eye diseases, including his company’s initative EyeWant2Know.com.

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