When I started my Twitch channel in February, I wasn’t sure what to expect. I became quite familiar with the video game streaming platform through journalism, and finally worked up the courage to start. In the four months since then, I’ve learned about starting a small business, branding, design,…
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
On May 21, the world celebrated its ninth Global Accessibility Awareness Day, but accessibility awareness hasn’t yet risen to its deserved level of interest. The awareness day website shares a great picture of this initiative’s potential scale: there are about a billion people with visual, hearing, motor, or cognitive…
NS Pharma’s lead candidate viltolarsen safely and effectively increases dystrophin levels and promotes improvements in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, final data from a Phase 2 trial show. “As a pediatric neurologist who specializes in the treatment of DMD, I am encouraged by the dystrophin…
A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne muscular dystrophy (DMD), final study results show. ATL1102, developed by Antisense Therapeutics, was also effective at lowering the number of immune cells targeted by its mechanism of action, improving upper…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.
The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid regimen to more effectively slow lung function decline in boys and men with Duchenne muscular dystrophy (DMD), has completed enrollment, Santhera Pharmaceuticals announced. A planned analysis demonstrated the study, while not enrolled up to the…
Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of dystrophin — the protein lacking in Duchenne muscular dystrophy (DMD) — and improves motor abilities in boys with the disorder, according to updated data from a Phase 1b trial. “Based on the…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
Get regular updates to your inbox.