News

October 21, 2020October 21, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 14, 2020October 14, 2020

Eli-cel Gene Therapy Continues to Show Encouraging Results for Boys with CALD, Clinical Data Show

News

Data from the Starbeam Phase 2/3 trial continues to support the safety and effectiveness of the gene therapy candidate elivaldogene autotemcel (eli-cel, formerly known as Lenti-D) for the treatment of cerebral ... Read more

October 9, 2020October 9, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News, syndicated

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

October 7, 2020October 7, 2020

Approval of CALD Gene Therapy, Lenti-D, Under Review in Europe

News

The European Medicines Agency (EMA) accepted an application requesting approval of the gene therapy Lenti-D (elivaldogene autotemcel, or eli-cel) to treat cerebral adrenoleukodystrophy (CALD), its developer, Bluebird Bio, announced. If approved, the submission — in ... Read more

September 28, 2020September 28, 2020

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

News, syndicated

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they ... Read more

September 23, 2020September 23, 2020

Phase 1 Study of VK0214 as Potential Oral Treatment for ALD to Open

News

Viking Therapeutics is launching a Phase 1 clinical trial testing increasing doses of VK0214, its oral investigative molecule to treat adrenoleukodystrophy (ALD), against a placebo in healthy people. “We are excited ... Read more

September 16, 2020September 16, 2020

Activities Underway for Newborn Screening Awareness Month

News, syndicated

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. ... Read more

August 31, 2020August 31, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

August 24, 2020August 31, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 17, 2020August 17, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more