Bionest

The SMA Market: Assessing the Unknowns

Challenges and opportunities in the field of spinal muscular atrophy, including how new disease-modifying therapies are likely to change SMA’s course over time.

Bionest

Zolgensma Launch “On Track,” but Significant Challenges Remain

On July 18, Novartis told analysts its Zolgensma launch was “on track.” Despite the gene therapy’s $2.1 million price tag, the company states the launch is “one of the most successful launches from an access standpoint in rare diseases.” Novartis said it had reached coverage deals with more than 20 commercial insurance plans, covering approximately 40%...

Bionest

Highlights from AAN: Part 1

The Bionest team attended the American Academy of Neurology (AAN) in early May, where we heard exciting data presented in a number of neurological conditions. Among the highlights of this year’s conference:   Transformative Success in Spinal Muscular Atrophy   Spinal muscular atrophy (SMA) was a hot topic at AAN, with presentations of positive clinical...

Bionest

Neuromuscular Drug Development Summit

At the end of October, we attended the inaugural Neuromuscular Drug Development Summit. This new meeting brought together thought leaders from industry, academia, and the patient advocacy community to discuss scientific progress and issues affecting companies developing drugs for these mostly rare diseases.   Neuromuscular diseases — such as muscular dystrophies, motor neuron diseases, mitochondrial...