Rhythm said that the failed trial of Imcrivee suffered a high dropout rate, which impacted the primary endpoints. No new safety signals were observed with Imcrivee, and the safety profile was consistent with prior clinical studies and commercial experience. The most common treatment-emergent adverse events (TEAEs) included skin hyperpigmentation, injection site reactions, nausea, vomiting, and headache. There had been a very high drop out rate in the trial, the company confirmed during an investor call. As a result, when using post hoc analysis using last observation carried forward for missing values, Imcrivee achieved statistically significant and clinically meaningful BMI reductions in the modified intent-to-treat patient populations in the POMC/PCSK1 Hets (5.5%) and SRC1 (6.2%). Dr David Meeker, CEO of Rhythm Pharmaceuticals, said: “While we are disappointed the EMANATE substudies did not meet their primary endpoint, we are encouraged by compelling signals from additional analyses of the heterozygous POMC/PCSK1 and SRC1 substudies and learnings that sharpen our ability to identify true loss-of-function variants and inform the development of our next-generation MC4R agonists in rare genetically driven obesity indications.” ... Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strat A Phase III trial of Rhythm Pharmaceuticals’ Imcivree (setmelanotide) failed to meet its primary endpoints in four rare, genetically driven obesities of the MC4R pathway.

Original title: “Rhythm’s Imcivree fails Phase III rare disease obesity trial - Clinical Trials Arena”

The Phase 3 EMANATE study is a basket trial looking at the efficacy of Rhythm Pharmaceuticals’ injectable obesity drug across four types of obesities driven by specific genetic mutations.

Original title: “Rhythm’s Imcivree Fails Late-Stage Weight Loss Trial in Rare Obesities”

rare cardiovascular diseases diagnostic progress and organizational gaps the belgian perspective

Original title: “Rare cardiovascular diseases: diagnostic progress and organizational gaps: the Belgian perspective.”

evaluation of mixed response in tumor size and survival in patients with rare cancers treated with dual checkpoint inhibitor therapy dart swog s1609

Original title: “Evaluation of mixed response in tumor size and survival in patients with rare cancers treated with dual checkpoint inhibitor therapy (DART SWOG S1609).”

Through creation of registries and repositories to increase access to small patient populations and few samples, innovative clinical trial designs that reduce participation burden, and unique therapeutic development approaches for personalized medicine, CDMRP-funded rare disease research overcomes ... In fiscal year 2026, the CDMRP received congressional appropriations to address rare cancers and several rare diseases and conditions with potential to impact Service Members during or after service, or their Family members, impacting military readiness. Through creation of registries and repositories to increase access to small patient populations and few samples, innovative clinical trial designs that reduce participation burden, and unique therapeutic development approaches for personalized medicine, CDMRP-funded rare disease research overcomes clinical challenges and accelerates treatment development. Developing treatments for rare diseases means working with small and globally dispersed patient populations and limited data, requiring highly creative and collaborative teams to identify and test new interventions. The CDMRP supports research in unique treatment approaches and innovative clinical trial designs for rare diseases, overcoming these challenges and accelerating treatment development for patients with few options. “This has been invaluable to bring the therapy closer to clinic.” · Oro’s work also demonstrates potential for translation to other types of wounds, including combat wounds experienced on the battlefield. “While the therapy focuses on helping the rare disease patients, it has broader impacts to heal poorly closing wounds for civilians, Service Members and Veterans due to injury or degeneration,” Oro said. “We believe this [study] has created a framework that can now be scaled to many other rare cancers, resources that empower labs everywhere, and the starting points for therapeutic trials that civilians, Service Members and Veterans can participate in,” Boehm said. Home-Based Strategies Increase Patient Participation in Epidermolysis Bullosa Clinical Trials

Original title: “DVIDS - News - CDMRP Funds Research to Accelerate Treatments for Rare Diseases to Support Warfighters and their Families”

On track to complete dosing in Embolden™ registrational trial of ... NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the · U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to · NGN-401, an investigational gene therapy in late-stage clinical development as a potential best-in-class treatment ... On track to complete dosing in Embolden™ registrational trial of ... NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the · U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to · NGN-401, an investigational gene therapy in late-stage clinical development as a potential best-in-class treatment for Rett syndrome. NGN-401 is the latest of several regulatory designations granted by FDA, including Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations, and selection for FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and · NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the NGN-401; the potential for success of the Embolden registrational clinical trial of · NGN-401 for Rett Syndrome; the clinical benefit of delivering · NGN-401 via intracerebroventricular administration; and expected future interactions with or positions of the FDA, including the timing and outcome of any such interaction and anticipated benefits of any regulatory designation for · NGN-401, including the FDA's Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation, and participation in the FDA's START program.

Original title: “Neurogene gets FDA Breakthrough Therapy nod for NGN-401 | NGNE Stock News”

Following the transfer of the IND for giroctocogene fitelparvovec (formerly SB-525, now PF-07055480), we initiated our Phase 3 trial evaluating giroctocogene fitelparvovec gene therapy for the treatment of severe hemophilia A. The future is here. By digging deeper, asking bold questions, and leading scientific innovation, Pfizer Rare Disease is striving to go beyond disease control toward developing potentially transformative medicines and supporting healthy living at all stages of life.

Original title: “Rare Disease Drug Pipeline and Clinical Trials | Pfizer”

Rare Diseases Clinical Research Network’s Coalition of Patient Advocacy Groups (RDCRN-CPAG) The Children’s Inn at NIH · EveryLife Foundation for Rare Diseases ·

Original title: “Rare Disease Day at NIH | National Center for Advancing Translational Sciences”

This observance, celebrated on the <strong>last day of February each year</strong>, highlights the importance of research and the development of new treatments for rare diseases, which often lack the attention and resources more common conditions receive.

Original title: “Rare Disease Day: How Patient Advocacy Groups Impact Research”

Angela is the Founder and President ... disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus that now includes 25% of the State Legislature. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the Rare Disease Legislative Advocates. In 2019, she was named a Top 100 Social Media Advocate for Rare Disease and in 2020, a Top 50 Rare Disease Awareness Advocate by the International Pain Foundation...

Original title: “Rare Disease Week - EveryLife Foundation for Rare Diseases”

Rare disease drug developers came ... of the year—Amgen’s acquisition of rare disease focused Horizon Therapeutics for <strong>$27.3 billion</strong>....

Original title: “Rare Disease Drug Developers Struggle Through a Difficult Finance Environment in 2022 - Global Genes”

Two significant acquisitions ushered in 2023 for rare disease drug developers with Chiesi Farmaceutici’s acquisition of Amryt Pharma for up to $1.5 billion and Ipsen’s acquisition of Albireo for up to $1.2 billion.

Original title: “Rare Disease Therapeutics M&A off to a Strong Start in 2023, But Financings Remain Lackluster - Global Genes”

By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood dis | By signing a government funding bill that ended a partial shutdown, President Donald Trump ... By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood dis | By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood diseases. Congress has struggled over the last year to bring back the beloved program, which initially expired at the end of 2024. “The passage of the Mikaela Naylon Give Kids a Chance Act is a bipartisan victory for children and families living with rare diseases,” John Crowley, president and CEO of the Biotechnology Innovation Organization, said in a Feb. The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon Give Kids a Chance Act and reviving the rare pediatric disease (RPD) priority review voucher program. The program will now be funded through September 2029.

Original title: “FDA’s rare pediatric disease voucher program revived by 2026 government funding bill”

Jan 28 (Reuters) - Regenxbio (RGNX.O), ... for rare childhood diseases, sending its shares plummeting 30% premarket. The company's experimental gene therapy RGX-111 was placed on clinical hold after a brain tumor was found in one trial participant, the company said. Sign up here. The tumor was discovered during a routine brain scan of a five-year-old child who had received the treatment about four ... Jan 28 (Reuters) - Regenxbio (RGNX.O), opens new tab said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its experimental gene therapy programs for rare childhood diseases, sending its shares plummeting 30% premarket. The company's experimental gene therapy RGX-111 was placed on clinical hold after a brain tumor was found in one trial participant, the company said. Sign up here. The tumor was discovered during a routine brain scan of a five-year-old child who had received the treatment about four years earlier, the company said. Regenxbio said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its experimental gene therapy programs for rare childhood diseases, sending its shares plummeting 30% premarket. RGX-111 is being developed as a treatment for Hurler syndrome, a rare autosomal recessive lysosomal storage disorder. It is estimated to occur in 1 in 100,000 births. Another therapy, RGX-121, being developed as a one-time treatment of MPS II or Hunter Syndrome, was also put on clinical hold by the FDA citing similarities between the two programs and a potentially shared risk. The company said it has not yet received the full clinical hold letter and awaits additional details from the FDA.

Original title: “US FDA places clinical hold on Regenxbio's rare disease program | Reuters”