Batten Disease News

September 17, 2021September 17, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

Batten Disease News

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 16, 2021September 16, 2021

FDA OKs Trial of NGN-101 Gene Therapy in CLN5 Patients

Batten Disease News

The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s request to conduct a Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of NGN-101, an investigational gene ... Read more

September 10, 2021September 10, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Batten Disease News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 9, 2021September 9, 2021

Neurogene Natural History Study Still Enrolling CLN5 Patients

Batten Disease News

A natural history study in people with CLN7 disease is now fully enrolled, but patients with CLN5 disease — each a form of late-infantile Batten disease, and both are in this study ... Read more

September 2, 2021September 2, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Batten Disease News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 26, 2021August 26, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Batten Disease News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 23, 2021August 26, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Batten Disease News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 19, 2021August 19, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Batten Disease News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 12, 2021August 12, 2021

Twins’ Different Symptoms Complicate Diagnosis

Batten Disease News

Juvenile Batten disease is difficult to diagnose promptly and accurately, as demonstrated by the case of twin girls with different symptoms of the same disorder. The case suggests that a ... Read more

August 12, 2021August 12, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Batten Disease News, COVID-19 News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more

August 5, 2021August 5, 2021

Advocates Lobby US Congress During Virtual Rare Disease Week

Batten Disease News, COVID-19 News

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the ... Read more

August 5, 2021August 5, 2021

LX1004 Gene Therapy Wins Orphan Drug, Rare Pediatric Disease Status

Batten Disease News

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to LX1004, an investigational gene therapy for CLN2 disease, also known as late ... Read more

July 29, 2021July 29, 2021

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

Batten Disease News

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other ... Read more

July 29, 2021July 29, 2021

Animal Models Can Predict Brineura Doses for CLN2 Patients

Batten Disease News

Animal models predicted dosing of the enzyme replacement therapy Brineura (cerliponase alfa) for patients with late infantile Batten disease, also called CLN2 disease, a study showed. Scientists said this approach ... Read more

July 22, 2021July 22, 2021

New Eye Exam Shows Promise as Vision Test for Children With CLN2

Batten Disease News

A new eye exam proved useful for testing vision in children with CLN2 disease — also known as late-infantile Batten disease — who took part in a pilot study, its ... Read more