South San Francisco-based Annexon, focused on drugs for autoimmune and neurodegenerative disorders, closed on a $100 million Series C financing led by Redmile Group.

Cambridge, Massachusetts-based Carmine Therapeutics inked a research collaboration deal with Japan’s Takeda Pharmaceutical to develop and commercialize non-viral gene therapies for two rare diseases.

The U.S. Food and Drug Administration approved Crysvita (burosumab) for the treatment of a rare disease, tumor-induced osteomalacia.

Lassen Therapeutics, based in San Diego, came out of stealth mode with a $31 million Series A financing round.

Cambridge, Massachusetts-based Alnylam Pharmaceuticals released positive Phase III data from the ILLUMINATE-A clinical trial of lumasiran in the treatment of primary hyperoxaluria type 1 (PH1).

Zolgensma’s successful market uptake by payers may hold lessons for other manufacturers of costly gene therapies.

The COVID-19 virus has fundamentally changed how we go about our daily lives on both a personal and professional level. We are becoming more accustomed to social distancing and virtual interaction with our friends, family members, loved ones, and colleagues. The change in behavior seems to be an easier adjustment in our personal lives than professional lives, but both have been impacted as a result of the virus.

When patients with a rare disease fail to receive a proper diagnosis, treatment plan, and therapy, they can suffer for years. Failed treatments for these prolonged conditions strain patients, caregivers, and the health ecosystem. In addition to the significant physical and emotional struggles these patients endure, delayed diagnoses for rare diseases can also result in many years of lost revenue for manufacturers.

Med Ad News talked to HVH Precision Analytics, a joint venture with Havas Health & You and Vencore specializing in AI and machine learning data analytics, about the company’s efforts at the forefront of rare disease work.

Charles Rosenberg, a historian of medicine and science, observed that viral outbreaks often unfold like a play in three acts. Act 1 is to ignore the mounting threat until it can’t be avoided. Act 2 is a call to arms (citizens demanding an explanation). And Act 3 – the one we are in now – is the response. Act 3, according to Rosenberg, can be “as disruptive as the disease itself.” The potential disruptions from Act 3 of COVID-19 have significant short- and long-term implications. Our proactive responsiveness to those disruptions can help drive success.