News

May 29, 2020May 29, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

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 ... Read more

May 28, 2020May 30, 2020

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

News

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 ... Read more

May 26, 2020May 26, 2020

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

News

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, ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

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 ... Read more

May 22, 2020May 22, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

cystic fibrosis, News, syndicated

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 ... Read more

May 21, 2020May 21, 2020

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

News

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 ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

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. ... Read more

May 19, 2020May 19, 2020

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

News

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 ... Read more

May 18, 2020May 18, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

May 14, 2020May 14, 2020

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

News

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the ... Read more

Pin It on Pinterest