Topic
rare diseases
22 stories related to this topic, newest first.
foxnews.comscience8 days ago
Blood Cancer United Buys Remaining Luvelta Supplies After Sutro Discontinued Development
The nonprofit will acquire the drug's investigational designation and run a no-cost compassionate-use program for children with a rare blood cancer after Sutro Biopharma ended development.
medpagetoday.comscience8 days ago
Mitapivat Cuts Transfusions but Misses Main Pain-Crisis Goal in Sickle-Cell Trial
Agios Pharmaceuticals presented new Phase 3 data showing mitapivat reduced transfusion burden and raised hemoglobin in sickle cell disease patients. The results were unveiled at the European Hematology Association Congress in Stockholm.
thehindubusinessline.comscience11 days ago
Servier Partners with n-Lorem Foundation on Preclinical Antisense Oligonucleotide Research for Rare Neurodevelopmental Disorders
The companies announced a multi-target research deal on June 10, 2026, to develop antisense oligonucleotide therapies for rare genetic neurological conditions.
New York Postscience30 days ago
Toddler Diagnosed with Sanfilippo Syndrome After Ear Infections and Developmental Regression
Cody Carroll began experiencing recurring ear infections and hearing loss at age two. He later received a diagnosis of Sanfilippo syndrome, a rare inherited disorder that leads to progressive neurological decline.
Statscience32 days ago
UCSF Team Submits Application for First In Utero Gene Therapy Trial
A University of California San Francisco team has submitted an investigational new drug application to the Food and Drug Administration for a small trial of in utero gene therapy. The proposed study would treat five fetal patients with a rare lysosomal storage disorder.
ncbi.nlm.nih.govworld34 days ago
Krystal Biotech Develops Gene Therapy for Rare Skin Disorder
Suma Krishnan, founder and president of Krystal Biotech, discussed a gene therapy treatment for epidermolysis bullosa at an America 250 event. The therapy targets the rare disease that leaves children born without fully formed skin.
FO
Statscience38 days ago
Regenxbio Gene Therapy for Duchenne Muscular Dystrophy Succeeds in Trial
Regenxbio announced on May 14, 2026 that its experimental treatment produced high levels of a miniaturized muscle protein, clearing the path for an FDA submission. The Rockville, Md.-based company aims to offer a safer and more effective option than Sarepta Therapeutics’ Elevidys…
The Independentworld38 days ago
NHS Approves Two Treatments for Spinal Muscular Atrophy
The National Institute for Health and Care Excellence has approved nusinersen and risdiplam for routine NHS use in England. The treatments were previously available only through a special access scheme. Muscular Dystrophy UK reports that the therapies have enabled 73 children wit…
prnewswire.comscience39 days ago
Encoded Therapeutics Reports Initial Results from Gene Therapy Trial for Dravet Syndrome
A trial by Encoded Therapeutics involving 21 children with Dravet syndrome showed that sirolimus, used alongside steroids to prevent immune responses, could blunt the therapy's benefits. The findings, published May 13, 2026, come from a Boston-based report examining a key concern…
prnewswire.combusiness40 days ago
Capsida Biotherapeutics Updates Investigation Into Death of Child in CAP-002 Gene Therapy Trial
The company said Tuesday it remains unable to determine what caused the death of a child last September in a trial of its experimental brain-targeted therapy CAP-002. Scientists have been blocked from full investigation because the hospital has declined to provide autopsy tissue…
pharmaphorum.comscience41 days ago
Astellas to Restart Gene Therapy Trial for XLMTM Five Years After Patient Deaths Halted Program
Patients and families affected by the rare muscle disorder XLMTM are receiving a second chance at treatment as Astellas relaunches its gene therapy program. The move comes as fresh turmoil grips the top of the FDA with questions swirling over whether Commissioner Marty Makary wil…
Statscience41 days ago
Parents of Boy with Rare Disease Report Reduced Suctioning Needs After NIH Treatment
Jessica Gonzalez realized an hour had passed without suctioning her son Joshua Jacob Gonzalez’s mouth and he was breathing normally, weeks after treatment at the National Institutes of Health. For his first two years, Javier and Jessica Gonzalez had suctioned saliva from the back…
Statscience44 days ago
Trump Administration Announces Projected Savings From Drug Pricing Agreements
The Trump administration is touting massive projected savings from still-secret drug pricing deals even as outside experts question how independently verifiable the figures are. STAT reported on the claims alongside fresh accusations of political interference at the FDA, which wi…
The Independentworld44 days ago
NHS England to Roll Out Givinostat for Eligible Duchenne Muscular Dystrophy Patients
Givinostat will become available to around 530 eligible patients in England with Duchenne muscular dystrophy following a commercial agreement between its manufacturer and NHS England. The National Institute for Health and Care Excellence confirmed the decision after the company r…
finance.yahoo.comscience46 days ago
FDA Shifts Remain Focus for Cell and Gene Therapy Industry at European Meeting
Cell and gene therapy developers gathered in Rome last week to discuss the sector's outlook in Europe. Conversations centered on recent FDA actions including a doubled rejection rate for such therapies since 2024 and the departure of the agency's top regulator for gene and cell t…
The Bbcworld46 days ago
Families Call for More Research into Progressive Supranuclear Palsy
Families of people diagnosed with progressive supranuclear palsy described the challenges of living with the rare neurological condition and called for greater awareness and research. The disease affects about 5,000 people in the UK and is often initially misdiagnosed as dementia…
insidermonkey.comscience70 days ago
Call to Address Structural Barriers in Rare Disease Treatments Affecting Over 300 Million People
A report highlights rare diseases impacting more than 300 million individuals worldwide. It advocates for removing structural obstacles in treatment development. The piece proposes viewing certain therapies as molecular surgery rather than traditional drug treatments.
ER
Substrate placeholder — needs reviewscience51 days ago
FDA Withdraws Approval for GSK's Leucovorin in Treating Cerebral Folate Deficiency
The U.S. Food and Drug Administration is withdrawing approval for GlaxoSmithKline's leucovorin as a treatment for cerebral folate deficiency, a rare brain disorder. GSK stated that the withdrawal was anticipated as part of the original approval plan. The disorder involves symptom…
ST
Substrate placeholder — needs reviewworld73 days ago
Recent Advances in Gene Therapy for Rare Diseases Reported
Developments in gene therapy and targeted medications have shown progress in treating rare diseases such as spinal muscular atrophy and certain forms of blindness. These conditions affect an estimated 30 million people in the United States, with over 7,000 identified rare disease…
Substrate placeholder — needs reviewscience74 days ago
Elevidys Gene Therapy Shows Positive Results in Duchenne Muscular Dystrophy Trial
Sarepta Therapeutics reported positive topline results from a phase 3 trial of its gene therapy Elevidys for Duchenne muscular dystrophy. The trial met its primary endpoint of improved motor function in patients aged 4 to 7. The Duchenne muscular dystrophy field has experienced s…
ST
Substrate placeholder — needs reviewscience74 days ago
Recent Health News Highlights Breakthrough in Duchenne Treatment and Insulin Price Cap
A new therapy shows promise for Duchenne muscular dystrophy patients. Insulin prices are capped at $35 for many users. Additional evidence supports over-the-counter availability of abortion medications.
ST
Substrate placeholder — needs reviewworld76 days ago
Neurocrine Biosciences Agrees to Acquire Soleno Therapeutics for $2.9 Billion
Neurocrine Biosciences has entered an agreement to purchase Soleno Therapeutics for $2.9 billion in cash. The acquisition aims to broaden Neurocrine's portfolio in treatments for rare genetic disorders. Soleno's lead product, DCCR, targets Prader-Willi syndrome, a rare condition…
BU