News

FLT190, Freeline Therapeutics’ experimental gene therapy, led to sustained increases in alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first two patients given the one-time therapy in the Phase 1/2 MARVEL-1 clinical trial. Notably, the second patient, dosed early this year, has reached near-normal…

A single dose of Avrobio’s investigational gene therapy AVR-RD-01 continues to show a favorable safety and tolerability profile for up to 4.5 years in adolescents and adults with Fabry disease, according to latest data from Phase 1 and Phase 2 clinical trials. Notably, up to 3.5-year data previously showed the…

Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in patients, updated results from the MODIFY Phase 3 trial show. The findings mean that Idorsia‘s MODIFY study (NCT03425539) did not meet its primary goal, despite lucerastat being well…

A Phase 1/2 clinical trial of Sangamo Therapeutics’ experimental gene therapy ST-920 in people with Fabry disease has completed dosing in second patient group, the company announced in a press release. Based on initial safety data, a trial monitoring committee has recommended that a planned third patient…

A recent type A meeting with the U.S. Food and Drug Administration (FDA) is likely to lead Protalix Biotherapeutics to resubmit a request for approval of PRX-102 (pegunigalsidase alfa) to treat Fabry disease, after its initial application was rejected by the agency. “We are pleased with the results of the…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

An eight-week, online mindfulness series for people with Fabry disease — aimed at relieving pain, reducing stress, and improving overall well-being — will open Sept. 29 and is currently enrolling participants. The Zoom series, which runs through Nov. 24, is presented by the MPS Society, which provides…